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P T. 2017;42(3): 155-157,160-164
New Drugs/Drug News

Drug and Device News March 2017

NEW DRUG APPROVALS

Advair and Flovent Copies for Asthma

AirDuo RespiClick (fluticasone propionate and salmeterol inhalation powder) and ArmonAir RespiClick (fluticasone propionate inhalation powder) have been approved by the FDA for the treatment of adolescents and adults with asthma. The two products, developed by Teva Pharmaceuticals, deliver medication via the RespiClick breath-activated, multi-dose dry-powder inhaler.

AirDuo RespiClick is a fixed-dose combination product containing the same active ingredients as Advair (Glaxo-SmithKline)—fluticasone propionate (a corticosteroid) and salmeterol (a long-acting beta2-adrenergic agonist)—but it delivers a lower dose of salmeterol. It also uses Teva’s RespiClick inhaler rather than a copy of GSK’s device.

ArmonAir RespiClick is an inhaled corticosteroid containing the same active ingredient as Flovent (Glaxo-Smith-Kline)—fluticasone propionate.

Source: Teva, January 30, 2017

Trulance for Constipation

The FDA has approved plecanatide (Trulance, Synergy Pharmaceuticals), a uroguanylin analogue, for the treatment of adults with chronic idiopathic constipation. Plecanatide, taken orally once daily, works locally in the upper gastrointestinal tract to stimulate the secretion of intestinal fluid and support regular bowel function. The safety and efficacy of plecanatide have not been established in patients younger than 18 years of age.

Source: Synergy Pharmaceuticals, January 19, 2017

Rhofade Cream For Facial Erythema

Oxymetazoline hydrochloride cream, 1% (Rhofade, Allergan), has received FDA approval for the topical treatment of persistent facial erythema associated with rosacea in adults. The product will be available in the United States in May 2017.

The FDA’s approval was based on data from two identical randomized, double-blind, parallel-group, vehicle-controlled studies involving a total of 885 patients with moderate or severe disease who were treated with oxymetazoline cream or vehicle. In study 1, the proportions of patients with reduced erythema at hours 3, 6, 9, and 12 on day 29 were 12%, 16%, 18%, and 15%, respectively, for oxymetazoline cream (n = 222) compared with 6%, 8%, 6%, and 6% for vehicle (n = 218). In study 2, the corresponding values were 14%, 13%, 16%, and 12% for oxymetazoline cream (n = 224) compared with 7%, 5%, 9%, and 6% for vehicle (n = 221).

Source: Allergan, January 19, 2017

Vantrela ER for Severe Pain

The FDA has given the “thumbs up” to Vantrela ER (extended-release hydrocodone bitartrate tablets [CII], Teva Pharmaceuticals), formulated with Teva’s proprietary abuse-deterrence technology. Vantrela ER is indicated for the management of pain that is severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate. The product’s abuse-deterrent properties are expected to reduce, but not totally prevent, oral, intranasal, and intravenous abuse of the drug when the tablets are manipulated.

Source: Teva, January 18, 2017

Arymo ER Morphine Tablets

The FDA has approved Arymo ER (extended-release morphine sulfate tablets [CII], Egalet Corporation) for the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate. The product has been approved in three dosage strengths: 15 mg, 30 mg, and 60 mg. The U.S. commercial launch is planned for the first quarter of 2017.

Arymo ER uses proprietary Guardian technology—a physical and chemical barrier approach to abuse deterrence without the use of an opioid antagonist. The technology creates tablets that are difficult to manipulate for the purpose of misuse or abuse.

Source: Egalet Corporation, January 9, 2017

Generic Approvals Sodium Oxybate Oral Solution

The FDA has given the green light to the first generic version of Jazz Pharmaceuticals’ Xyrem (sodium oxybate) oral solution to treat cataplexy and excessive daytime sleepiness in patients with narcolepsy, a potentially debilitating disease. The generic product was developed by Roxane Laboratories. Cataplexy is a primary symptom of narcolepsy in which patients suddenly lose muscle tone, including voluntary muscle control, while awake.

Source: FDA, January 17, 2017

Desoximetasone Topical Spray, 0.25%

Perrigo Company has received FDA approval for its abbreviated new drug application referencing Topicort spray, 0.25% (desoximetasone topical spray, 0.25%, Taro Pharmaceuticals). Prior patent litigation between the companies has been settled on confidential terms. Desoximetasone topical spray, 0.25%, is a corticosteroid indicated for the treatment of plaque psoriasis in patients 18 years of age or older.

Source: Perrigo Company, January 24, 2017

Bendamustine Hydrochloride Powder for Intravenous Infusion

The FDA has granted Breckenridge Pharmaceutical, Inc., final approval of its abbreviated new drug application for bendamustine hydrochloride powder for intravenous infusion, 25 mg/vial and 100 mg/vial—a generic version of Treanda (Cephalon, Inc.). Treanda is indicated to treat patients with chronic lymphocytic leukemia and non-Hodgkin’s lymphoma. Breckenridge intends to launch the product by November 1, 2019.

Source: Breckenridge Pharmaceutical, January 9, 2017

Ephedrine Sulfate Injection

Par Pharmaceutical, a subsidiary of Endo International, has received final approval from the FDA for its new drug application for ephedrine sulfate injection, a drug administered parenterally as a pressor agent to treat clinically important hypotension in surgical settings. The product is packaged in cartons of 25 50-mg/mL, 1-mL, single-use vials. Par projected that shipments would begin in February 2017.

Source: Endo International, January 30, 2017

NEW INDICATIONS

Imbruvica for Marginal-Zone Lymphoma

The FDA has approved ibrutinib (Imbruvica, Janssen Biotech/AbbVie) for the treatment of patients with relapsed/refractory marginal-zone lymphoma (MZL) who require systemic therapy and have received at least one prior anti–CD20-based therapy. In the U.S., ibrutinib now has five distinct indications: chronic lymphocytic leukemia, small lymphocytic lymphoma, Waldenström’s macroglobulinemia, previously treated mantle-cell lymphoma, and MZL.

The FDA’s decision was based on data from a phase 2, open-label, multicenter, single-arm study involving 63 patients with the three subtypes of MZL: mucos-aassociated lymphoid tissue (n = 32), nodal (n = 17), and splenic (n = 14). The overall response rate was 46%, with efficacy observed across all three MZL subtypes. The median period to a response was 4.5 months (range, 2.3–16.4 months). In the study, 3.2% of patients had a complete response, and 42.9% had a partial response.

Source: AbbVie, January 19, 2017

Latuda for Adolescents With Schizophrenia

The FDA has approved a supplemental new drug application for lurasidone (Latuda, Sunovion Pharmaceuticals), an atypical antipsychotic, for the treatment of schizophrenia in adolescents 13 to 17 years of age. Lurasidone was previously indicated in the U.S. for the treatment of adults with schizophrenia and for the treatment of adults with major depressive episodes associated with bipolar I disorder (bipolar depression) as monotherapy and as adjunctive therapy with lithium or valproate.

Source: Sunovion Pharmaceuticals, January 28, 2017

Symbicort for Pediatric Asthma Patients

Symbicort (budesonide/formoterol fumarate dihydrate) inhalation aerosol 80/4.5 mcg (AstraZeneca) has been approved by the FDA for the treatment of asthma in pediatric patients 6 to 12 years of age. Symbicort (80/4.5 mcg and 160/4.5 mcg) is already approved in the U.S. to treat asthma patients 12 years of age and older. Symbicort 160/4.5 mcg is also approved for the maintenance treatment of airflow obstruction in adults with chronic obstructive pulmonary disease.

Symbicort is an inhaled corticosteroid/long-acting beta2-agonist bronchodilator controller medication that has been shown to help control asthma by reducing inflammation in the lungs, thereby keeping airways open. The product is not indicated for the relief of acute bronchospasm.

Source: AstraZeneca, January 30, 2017

NEW FORMULATION

Narcan Nasal Spray 2 mg For Opioid Overdose

The FDA has given the nod to Narcan nasal spray (naloxone, Adapt Pharmaceuticals) as a 2-mg formulation for the emergency treatment of known or suspected opioid overdose, as manifested by respiratory and/or central nervous system depression. The 2-mg dose is approved for use in opioid-dependent patients expected to be at risk for severe opioid withdrawal in situations where there is a low risk for accidental or intentional opioid exposure by household contacts. This approval comes approximately one year after the FDA approved the first ready-to-use, needle-free version of naloxone, Narcan nasal spray 4 mg, which has been commercially available since February 2016.

Source: Adapt Pharmaceuticals, January 25, 2017

FDA REVIEW ACTIVITIES

Priority Review Designations

Actemra for Giant Cell Arteritis

The FDA has accepted a supplemental biologics license application for tocilizumab (Actemra/RoActemra, Roche) for the treatment of patients with giant cell arteritis (GCA), a chronic, potentially life-threatening autoimmune condition. The agency also granted priority review status to tocilizumab for GCA. This designation was based on positive results from the phase 3 GiACTA study, in which tocilizumab, initially combined with a six-month steroid (glucocorticoid) regimen, more effectively sustained remission through one year compared with a six- or 12-month steroid-taper regimen given alone in patients with GCA. Tocilizumab is the only approved anti–interleukin-6 receptor biologic for the treatment of adults with moderately or severely active rheumatoid arthritis.

Source: Roche, January 24, 2017

Tecentriq for Advanced Bladder Cancer

After accepting the supplemental biologics license application for atezolizumab (Tecentriq, Genentech), the FDA has granted the product priority review for the treatment of patients with locally advanced or metastatic urothelial carcinoma who are ineligible for cisplatin chemotherapy, and who are either previously untreated (first-line) or have disease progression at least 12 months after receiving chemotherapy before surgery (neoadjuvant) or after surgery (adjuvant). The FDA will make a decision on approval by April 30, 2017.

Atezolizumab is a monoclonal antibody designed to bind with the programmed death ligand-1 (PD-L1) protein on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both programmed death-1 (PD-1) and B7.1 receptors. By inhibiting PD-L1, atezolizumab may enable the activation of T cells. The antibody may also affect normal cells.

Source: Genentech, January 8, 2017

Fast-Track Designation

Resolaris for Muscular Dystrophy

Resolaris (aTyr Pharma), a potential first-in-class intravenous protein therapeutic, has received a fast-track designation from the FDA for the treatment of patients with limb girdle muscular dystrophy 2B (LGMD2B). In addition, the FDA removed its partial clinical hold on a dosing ceiling for Resolaris in clinical studies.

A phase 1b/2 open-label, intrapatient, dose-escalation trial tested doses of Resolaris of up to 3.0 mg/kg biweekly in nine patients with LGMD2B. Seven of the patients (78%) recorded increases in their muscle function at 14 weeks, as measured by the manual muscle test (MMT). Overall, the patients had a mean increase in MMT scores from baseline of 6.2%.

Source: aTyr Pharma, January 18, 2017

Label Update

Avycaz Phase 3 Data

The FDA has allowed the label for Avycaz (ceftazidime/avibactam, Allergan/Pfizer) to be updated with clinical data from two phase 3 trials supporting the medication’s indication to treat patients with complicated urinary tract infections, including pyelonephritis, caused by designated susceptible gram-negative microorganisms.

In the pivotal multicenter, double-blind RECAPTURE trial, the symptomatic response rate at day 5 in Avycaz-treated patients was 70.2% (276/393) compared with 66.2% (276/417) in doripenem-treated patients, for a treatment difference of 4.0 percentage points. The combined symptomatic and microbiological response rate at the test-of-cure visit among Avycaz-treated patients was 71.2% (280/393) compared with 64.5% (269/417) among doripenem treated patients, for a treatment difference of 6.7 percentage points.

Source: Allergan, January 30, 2017

Orphan Drug Designations

GS030 for Retinitis Pigmentosa

GS030 (GenSight Biologics) has been awarded orphan drug status by the FDA for the treatment of patients with retinitis pigmentosa (RP). GS030 is expected to enter clinics with a phase 1/2 clinical trial in patients with RP in the third quarter of 2017, subject to toxicity results and future regulatory review.

Source: GenSight Biologics, January 31, 2017

TG-1101/TGR-1202 for Lymphoma

The FDA has granted an orphan drug designation covering the combination of TG-1101 (ublituximab), a glycoengineered anti-CD20 monoclonal antibody, and TGR-1202, an oral, next-generation phosphoinositide 3-kinasedelta inhibitor, for the treatment of patients with diffuse large B-cell lymphoma (DLBCL). Both compounds were developed by TG Therapeutics.

The combination of TG-1101 and TGR-1202 is being evaluated in the phase 2b UNITY-DLBCL trial in patients with relapsed or refractory DLBCL as well as in the phase 3 UNITY-CLL trial in patients with both front-line and previously treated chronic lymphocytic leukemia.

Source: TG Therapeutics, January 24, 2017

Viralym-C for CMV Infection

Viralym-C (ViraCyte), a T-cell immunotherapy product for the treatment of cytomegalovirus (CMV) infection in severely immunocompromised patients, has been granted an orphan drug designation by the FDA. In January 2017, Viralym-C was also granted fast-track status for the same indication. In patients with weakened immune systems, CMV infection can cause severe disease manifestations, including pneumonia, and can be fatal.

Source: ViraCyte, January 24, 2017

AD-114 for Pulmonary Fibrosis

AdAlta Ltd. has received an orphan drug designation from the FDA for AD-114, a first-in-class drug candidate for the treatment of patients with idiopathic pulmonary fibrosis (IPF). AD-114 has demonstrated both antifibrotic and anti-inflammatory activity in human lung tissue, indicating greater efficacy than that of existing approved IPF drugs, according to the drug’s developer. Phase 1 clinical trials in subjects with IPF are expected to begin by early 2018.

Source: AdAlta, January 18, 2017

Rare Pediatric Disease Designation

LYS-GM101 for Gangliosidosis

The FDA has granted a rare pediatric disease designation to LYS-GM101 (Lysogene), a drug candidate in development for patients with GM1 gangliosidosis, a neurodegenerative disorder. GM1 is an extremely severe, autosomal recessive disease caused by a mutation in the GLB1 gene encoding for the lyso-somal acid beta-balactosidase enzyme. The resulting enzymatic deficiency leads to the accumulation of GM1-ganglioside in cells. LYS-GM101 has the potential to replace the defective gene in the cells of GM1 patients, which allows production of the functional enzyme and prevents the progression of the neurological damage caused by GM1.

Source: Lysogene, January 18, 2017

Qualified Infectious Disease Product

PUR1900 for Lung Infection in CF Patients

The FDA has designated PUR1900 (itraconazole inhalation powder, Pulmatrix, Inc.) as a qualified infectious disease product (QIDP) for the potential treatment of pulmonary Aspergillus infection in patients with cystic fibrosis. Under the agency’s QIDP program, which is designed to speed the development of new drugs against important pathogens, Pulmatrix will receive five years of additional market exclusivity for PUR1900. The combination of itraconazole and dry-powder iSPERSE technology makes it possible for patients to inhale the drug into their lungs to the site of infection.

Source: Pulmatrix, January 17, 2017

New or Revised Drug Applications

Briviact for Epilepsy

UCB has filed a supplemental new drug application with the FDA for brivaracetam (Briviact) as monotherapy for partial-onset seizures in patients 16 years of age and older with epilepsy. Brivaracetam is currently approved in the U.S. as adjunctive therapy in the treatment of partial-onset seizures in patients 16 years of age and older with epilepsy. The most common adverse reactions in adjunctive therapy trials included somnolence, sedation, dizziness, fatigue, nausea, and vomiting.

Brivaracetam is selective for synaptic vesicle protein 2A in the brain, which may contribute to its anticonvulsant effect. However, the precise mechanism of action by which brivaracetam exerts its anticonvulsant activity is not known.

Source: UCB, January 19, 2017

SHP465 for ADHD

The FDA has acknowledged receipt of the class 2 resubmission of a new drug application (NDA) for SHP465 (Shire), a long-acting, triple-bead, mixed amphetamine salts formulation. The compound is being evaluated as a potential once-daily treatment for patients with attention-deficit/hyperactivity disorder (ADHD). The FDA is expected to provide an approval decision in June 2017. Shire resubmitted the NDA for SHP465 in response to a letter from the FDA (May 2007) that requested additional clinical studies and classified the response as a class 2 resubmission with a review goal of six months.

Source: Shire, January 19, 2017

Solosec for Bacterial Vaginosis

Symbiomix has submitted a new drug application to the FDA for Solosec (secnidazole oral granules) as a potential treatment for women with bacterial vaginosis (BV). Solosec is an investigational 5-nitroimidazole antibiotic with enhanced pharmacokinetic properties. If approved, it would be the first single-dose oral therapy for BV. The FDA has designated Solosec as a qualified infectious disease product for the treatment of BV and granted the drug a fast-track designation in 2015.

Source: Symbiomix, January 19, 2017

EZR-104 for Hypertension And Heart Failure

The FDA has accepted a new drug application (NDA) for EZR-104 (Ezra Innovations) for the treatment of patients with hypertension and heart failure. The Prescription Drug User Fee Act goal date for approval of the NDA is September 10, 2017.

EZR-104 combines the angiotensin receptor blocker valsartan with patented Rubireten delivery technology. This new formulation is designed to control the release of the active drug throughout the 24-hour dosing period and to overcome diminished blood concentrations of the drug when administered with a meal, an effect commonly associated with previously approved forms of valsartan.

Source: Ezra Innovations, January 18, 2017

Humira Biosimilar Candidate

BI 695501 (Boehringer Ingelheim), an adalimumab biosimilar candidate to Humira (AbbVie), has been accepted for regulatory review by the FDA. Adalimumab is a monoclonal antibody that blocks tumor necrosis factor-alpha, an important mediator of inflammation. Top-line results from a completed phase 3 study of BI 695501 in patients with active rheumatoid arthritis were announced in October 2016.

Source: Boehringer Ingelheim, January 18, 2017

Eptacog Beta for Hemophilia

The FDA has accepted a biologics license application for eptacog beta (coagulation factor VIIa [recombinant], HEMA Biologics) for patients with hemophilia A and B with inhibitors. The license application was supported by data from a pivotal phase 3 trial, which focused on the potential treatment of episodic bleeding events in adult and adolescent patients. Two initial dose regimens were tested in 468 bleeding events in 27 inhibitor patients. Both treatment arms met the primary endpoint of hemostatic success (i.e., evidence of bleeding cessation). Most (85%) of the bleeding events treated with the initial 225-mcg/kg dose required no further therapy.

Source: HEMA Biologics, January 9, 2017

Ingrezza for Tardive Dyskinesia

The FDA, after reviewing the new drug application (NDA) for valbenazine (Ingrezza, Neurocrine Biosciences), decided to cancel a meeting of its Psychopharmacologic Drugs Advisory Committee that was scheduled for February 16, 2017. The FDA informed Neurocrine, however, that the priority review of the NDA for valbenazine for the treatment of patients with tardive dyskinesia is continuing as planned, with the previously announced target action date of April 11, 2017.

Vesicular monoamine transporter 2 (VMAT2), a protein concentrated in the human brain, is primarily responsible for repackaging and transporting monoamines (dopamine, norepinephrine, serotonin, and histamine) in presynaptic neurons. Valbenazine is a highly selective VMAT2 inhibitor that modulates dopamine release during nerve communication, showing little or no affinity for VMAT1 or for other receptors, transporters, and ion channels.

Source: Neurocrine Biosciences, January 6, 2017

DRUG SAFETY ISSUES

Tecfidera and Liver Injury

The prescribing information for the popular multiple sclerosis drug Tecfidera (dimethyl fumarate, Biogen) has been updated to include a warning of potential liver injury that could require hospitalization. The new information can be found in a warning section under “prescribing information” on the drug’s website. The warning reads: “Clinically significant cases of liver injury have been reported in patients treated with Tecfidera in the post-marketing setting. The onset has ranged from a few days to several months after initiation of treatment with Tecfidera.” The signs of liver injury resolved when affected patients stopped taking the medication.

Source: Reuters, January 25, 2017

Adverse Effects of Alemtuzumab

The multiple sclerosis (MS) therapy alemtuzumab—marketed in the United States as Lemtrada (Genzyme Corporation)—may trigger severe, unpredictable adverse effects, according to German researchers. Writing in Lancet Neurology, the scientists reported on two patients in whom the infusion of alemtuzumab, an anti-CD52 antibody, significantly worsened symptoms.

The two patients had highly active MS despite numerous prior treatments and because they experienced severe relapses, with inflammation in the central nervous system. Six months after treatment with alemtuzumab, these symptoms worsened significantly. Using magnetic resonance imaging, the researchers discovered a new mode of inflammation: they found areas in the brain with numerous ring-enhancing lesions. The patients had not displayed this pattern in their previous medical histories. It was unclear, however, whether the observed adverse events represented increased MS activity or an independent secondary autoimmune process.

Source: Ruhr University Bochum, January 18, 2017

Catheter Safeguards Reduce Infections

A study led by investigators at the Cedars–Sinai Medical Center in Los Angeles, California, looked at data published during the last decade on catheter-related bloodstream infections at 113 hospitals. The study found that safety interventions, on average, reduced the infection rate by 57% at these hospitals while producing net savings of $1.85 million for each site over three years. The savings came from reduced costs in treating infected patients.

The report, published in JAMA Internal Medicine, focused on central venous catheters, which are commonly used in intensive care units. More than 60,000 primary bloodstream infections related to these catheters are estimated to occur each year in the United States. To prevent these infections, hospitals have introduced new safety procedures, including donning sterile gloves, covering catheters with antimicrobial dressings, and checking catheters daily for signs of movement or infection. Many hospitals have also invested in extra training, equipment, and supplies to improve safety.

In the hospitals studied, the median cost of implementing catheter safety programs was approximately $270,000 per site. But for every $100,000 that a hospital spent, it realized an average $315,000 savings because it treated fewer infected patients, the investigators found.

Source: Cedars–Sinai Medical Center, January 9, 2017

CLINICAL TRIAL NEWS

Edasalonexent for DMD

Catabasis Pharmaceuticals has announced disappointing results from part B of the MoveDMD trial of edasalonexent (CAT-1004) in patients with Duchenne muscular dystrophy (DMD). The objective of part B was to evaluate the effects of edasalonexent using magnetic resonance imaging (MRI) T2 as a biomarker at 12 weeks. The study failed to meet the primary efficacy endpoint of an average change from baseline to week 12 in the MRI T2 composite measure of lower leg muscles for the pooled edasalonexent treatment groups of 67 mg/kg per day and 100 mg/kg per day compared with placebo (0.37 msec for the pooled edasalonexent groups versus 0.47 msec for the placebo group). The open-label extension portion (part C) of the MoveDMD trial is ongoing.

Source: Catabasis Pharmaceuticals, January 31, 2017

Levosimendan in Cardiac Surgery

Mixed results have been reported from a phase 3 trial of levosimendan (Tenax Therapeutics) in patients with left ventricular systolic dysfunction undergoing cardiac surgery. The study did not achieve statistically significant reductions in the dual endpoint of death or the use of a mechanical assist device at 30 days, nor in the quad endpoint of death, myocardial infarction, the need for dialysis, or the use of a mechanical assist device at 30 days. However, the study demonstrated statistically significant reductions in two of the three secondary endpoints, including a reduction in low cardiac output syndrome and a reduction in the postoperative use of secondary inotropes. Levosimendan is a calcium sensitizer that was initially developed for intravenous use in hospitalized patients with acutely decompensated heart failure.

Source: Tenax Therapeutics, January 31, 2017

Lenvima for Liver Cancer

A phase 3 clinical study of the anticancer agent lenvatinib mesylate (Lenvima, Eisai) compared with sorafenib (Nexavar, Bayer) as a first-line treatment for patients with unresectable hepatocellular carcinoma has achieved its primary endpoint with the goal of demonstrating noninferiority. Lenvatinib is an orally administered multiple receptor tyrosine kinase inhibitor.

A total of 954 patients were randomly assigned to receive lenvatinib 8 mg or 12 mg once daily, depending on baseline body weight (n = 478), or sorafenib 400 mg twice daily (n = 476) until disease progression or unacceptable toxicity occurred. The study’s primary endpoint was overall survival (OS). Secondary endpoints included progression-free survival, the time to progression, and the objective response rate.

Lenvatinib met the statistical criteria for noninferiority of OS compared with sorafenib and showed statistically significant and clinically meaningful improvements in the three secondary endpoints.

Source: Eisai, January 25, 2017

BGB-3111 for Macroglobulinemia

BeiGene, Ltd., has initiated a global, randomized, phase 3 study of its investigational Bruton’s tyrosine kinase (BTK) inhibitor BGB-3111 in patients with Waldenström’s macroglobulinemia (WM). The study is designed to determine whether the quality of response with BGB-3111 in WM is superior to that of ibrutinib (Imbruvica, Pharmacyclics/Janssen Biotech), the currently approved BTK inhibitor. Approximately 170 patients are expected to be enrolled at clinical sites in North America, Europe, Australia, and New Zealand.

Source: BeiGene, January 25, 2017

Livatag for Liver Cancer

Enrollment has been completed in the phase 3 ReLive trial, an international study evaluating intravenous Livatag (doxorubicin transdrug, Onxeo S.A.) for the treatment of patients with advanced hepatocellular carcinoma. A total of 390 patients have been enrolled, with 260 patients in the Livatag group and 130 in the comparator group (best standard of care).

Livatag is a nanoparticle formulation of doxorubicin developed using proprietary transdrug technology, which is designed to facilitate the penetration of the drug into the tumor cell, thereby increasing the target DNA exposure to the drug and bypassing the mechanisms of multidrug resistance developed by tumor cells.

Source: Onxeo, January 24, 2017

Spinal Cord Stimulation For Chronic Pain

A new study has found that spinal cord stimulation (SCS) therapy may help reduce or stabilize the use of opioids in patients with chronic pain. Researchers examined opioid-use data from more than 5,400 patients before and after receiving an SCS implant (Abbott). In an SCS system, an implanted device similar to a pacemaker delivers low levels of electrical energy to nerve fibers, thereby interrupting pain signals as they travel to the brain to reduce the sensation of pain.

The researchers found that average daily opioid use declined or stabilized in patients receiving a successful SCS implant compared with opioid use before the implant. In addition, while opioid use was not different for the two groups at the time of implant, patients who underwent a successful SCS implant had significantly lower opioid use one year after their implant, whereas patients who had their SCS implant removed saw their opioid use increase over time.

Source: Abbott, January 20, 2017

Opdivo for Previously Treated Gastric Cancer

Positive results have been reported from a phase 3, randomized, placebo-controlled trial of nivolumab (Opdivo, Bristol-Myers Squibb/Ono Pharmaceuticals) in patients with previously treated advanced gastric cancer who were refractory to or intolerant of standard therapy. The study was conducted in Japan, Korea, and Taiwan.

Opdivo 3 mg/kg or placebo was administered every two weeks until disease progression or discontinuation due to unacceptable toxicity. The primary efficacy endpoint was overall survival (OS). The median OS was 5.32 months for nivolumab compared with 4.14 months for placebo (P < 0.0001). In addition, the 12-month OS in the nivolumab group was 26.6% compared with 10.9% in the placebo group. Patients treated with nivolumab also experienced an objective response rate of 11.2% compared with 0% for placebo, and a median duration of response of 9.53 months.

Source: Bristol-Myers Squibb, January 19, 2017

Cimzia for Plaque Psoriasis

Positive results have been reported from a pivotal phase 3 study of certolizumab pegol (Cimzia, UCB/Dermira, Inc.) in adults with moderate-to-severe chronic plaque psoriasis. Based on the findings from this study, UCB plans to submit a marketing application to the FDA in the third quarter of 2017. Certolizumab pegol is not currently approved for the treatment of psoriasis by any regulatory authority worldwide.

In the randomized, blinded, parallel-group, placebo- and active-controlled, multicenter CIMPACT trial, the response rates at week 12 for patients who achieved 75% or greater disease improvement from baseline, as measured by the Psoriasis Area and Severity Index, were 66.7% for those receiving certolizumab pegol 400 mg every two weeks and 61.3% for those receiving certolizumab pegol 200 mg every two weeks compared with 5.0% for patients receiving placebo.

Source: UCB, January 19, 2017

Defitelio for Liver Disease

The first patient has been enrolled in a phase 3 clinical trial comparing defibrotide (Defitelio, Jazz Pharmaceuticals) with best supportive care in the prevention of hepatic veno-occlusive disease (VOD)—also known as sinusoidal obstruction syndrome—in adult and pediatric patients undergoing hematopoietic stem cell transplant (HSCT) who are at high risk or very high risk of developing VOD. Defibrotide injection 80 mg/mL received FDA approval in March 2016 for the treatment of adult and pediatric patients with hepatic VOD with renal or pulmonary dysfunction after HSCT.

The randomized, open-label, multicenter study is expected to enroll approximately 400 patients, who will receive defibrotide starting on the day before the first day of the conditioning regimen for a recommended minimum of 21 days and ending no later than day 30+ post-HSCT. The study’s primary endpoint is VOD-free survival at day 30+ post-HSCT.

Source: Jazz Pharmaceuticals, January 18, 2017

AquaBeam System for BPH

Procept BioRobotics has announced the completion of patient enrollment into the global phase 3 WATER trial. This double-blind, prospective, randomized study involves men between 45 and 80 years of age who have urinary symptoms due to benign prostatic hyperplasia (BPH). The study is evaluating the safety and efficacy of the AquaBeam system compared with that of transurethral resection of the prostate. The study’s primary endpoints of safety and efficacy will be measured at three and six months, respectively, and patients will be followed for three years to collect long-term clinical data. The Aqua-Beam system combines real-time prostate imaging and surgical robotics to deliver Aquablation, a waterjet ablation therapy that enables targeted removal of prostate tissue for the treatment of lower urinary tract symptoms caused by BPH.

Source: Procept BioRobotics, January 10, 2017

IDP-118 for Plaque Psoriasis

Positive results have been reported from a pivotal phase 3, multicenter, double-blind, randomized, vehicle-controlled clinical study that assessed the safety and efficacy of IDP-118 (halobetasol propionate and tazarotene lotion, Valeant Pharmaceuticals) in the treatment of patients with plaque psoriasis. The study enrolled 203 patients with moderate-to-severe disease.

The primary endpoint of the 12-week study (eight weeks of treatment and four weeks of follow-up) was the achievement of a “clear” to “almost clear” score and at least a two-grade improvement based on an Investigator Global Assessment (IGA) at eight weeks. Secondary endpoints included “clear” to “almost clear” scores and at least a two-grade improvement in the IGA at weeks 2, 4, 6, and 12. IDP-118 was significantly more effective than vehicle at week 8, with a treatment success rate of 35% versus 7%, respectively (P < 0.001).

Source: Valeant, January 10, 2017

BrainPath Approach for Hemorrhagic Stroke

The first patient has been enrolled in the ENRICH trial, which is designed to determine the procedural safety as well as the economic and functional benefit of early surgical removal of intra-cerebral hemorrhage using the BrainPath approach (NICO Corporation) compared with standard-of-care medical management in a total of 300 patients. The Brain-Path approach involves a combination of two technologies, the FDA-cleared NICO BrainPath for nondisruptive access and NICO Myriad for maximum clot evacuation.

Source: NICO Corporation, January 10, 2017

DEVICE APPROVALS

GEO Bone Screw System

Gramercy Extremity Orthopedics has received FDA 510(k) clearance for its GEO Bone Screw System, which can be used for bone fractures, osteotomies, arthrodesis, osteochondritis, and tendon reattachments. The system offers an array of low-profile titanium screw lengths, diameters, thread lengths, and fully threaded options.

Source: Gramercy Extremity Orthopedics, January 26, 2017

Image Guidance System For Spine Surgery

7D Surgical, based in Toronto, Canada, has received 510(k) clearance from the FDA for the company’s 7D Surgical System, a machine-vision image-guided surgery (MIGS) system for spine surgery. The system employs 3D optical technologies and machine-vision algorithms to register spinal surgery patients automatically using only visible light. The MIGS navigation technology is embedded in an overhead surgical light, and the software is controlled by the surgeon using a foot pedal.

Source: 7D Surgical, January 23, 2017

Siconus SI Joint Fixation System

The FDA has granted 510(k) clearance to the Siconus SI joint fixation system (Camber Spine Technologies). The Siconus SI system is the second product in the company’s portfolio to treat sacroiliac disease, as a complement to the Prolix custom-machined allograft implant. The system is designed to provide fixation and stabilization of large bones, including the sacrum and ilium. It is intended for use in skeletally mature patients as an adjunct to sacroiliac joint fusion in the treatment of degenerative sacroiliitis or sacroiliac joint disruptions.

Source: Camber Spine Technologies, January 23, 2017

Pleur-X Catheter System For Pleural Effusions

The PleurX catheter system (Becton, Dickinson and Company) has received 510(k) clearance from the FDA for use in specific nonmalignant, recurrent pleural-effusion etiologies, including congestive heart failure. A pleural effusion is a collection of fluid that accumulates in the pleural cavity and surrounds the lungs, leaving patients short of breath and affecting their quality of life. When this occurs, the PleurX catheter system provides patients with the option to drain the pleural fluid in their homes, which can help avoid the need for multiple hospital visits for thoracentesis procedures.

Source: Becton, Dickinson and Company, January 23, 2017

OssDesign Cranial Implant

Swedish company OssDesign AB has received 510(k) clearance from the FDA for the marketing and sale of OssDesign Cranial PSI in the United States. The product is a patient-specific medical implant intended for the reconstruction of cranial defects (cranioplasty). Each implant is made to the patient’s unique requirements, based on his or her computed tomography information. The implant is made from a proprietary calcium phosphate composite, which is reinforced by a titanium skeleton.

Source: OssDesign, January 20, 2017

Bindex Device for Diagnosis of Osteoporosis

The FDA has granted 510(k) clearance to the second-generation Bindex (Bone Index, Ltd.) point-of-care device to help in the diagnosis of osteoporosis. The device measures the cortical bone thickness of the tibia, and the algorithm calculates the density index—a parameter that estimates bone mineral density at the hip, as measured with dual-energy x-ray absorptiometry. The Bindex device detects osteoporosis with 90% sensitivity and specificity, according to its manufacturer.

Source: Bone Index, Ltd., January 20, 2017

Troponin T Test

Roche has received 510(k) clearance for its Elecsys Troponin T Gen 5 STAT blood test to aid in the diagnosis of myocardial infarction. The assay detects cardiac troponin, which is the preferred biomarker for the diagnosis of heart attack in clinical practice. In combination with an electrocardiogram and signs of ischemia, troponin has become the gold standard as an aid in the diagnosis of heart attack. The higher sensitivity of this assay in conjunction with its rapid turnaround time of nine minutes can significantly accelerate decision-making and allows the detection of smaller infarctions, thereby maximizing the potential for effective treatment, according to Roche.

Source: Roche, January 19, 2017

Obalon Balloon System For Weight Loss

The FDA has approved the Obalon balloon system (Ultimate Bariatrics), the first ingestible, nonsurgical, fully reversible, six-month balloon system to help patients achieve their weight-loss goals. The system consists of three lightweight balloons, which are placed gradually over six months. The balloons occupy space in the patient’s stomach so that he or she feels full and less hungry, making weight loss easier. The treatment should be accompanied by a professionally supervised diet and exercise program.

Source: Ultimate Bariatrics, January 19, 2017

AirMini for CPAP

The FDA has cleared AirMini (ResMed, Inc.), the world’s smallest continuous positive airway pressure (CPAP) device. The product, to be launched later this year, is designed as a secondary CPAP device, making it easier for people to continue their sleep apnea therapy while traveling, according to the manufacturer.

Source: ResMed, January 9, 2017