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Drug and Device News May 2016
NEW DRUG APPROVALS
Venclexta for CLL
The FDA has approved venetoclax (Venclexta, AbbVie/Genentech) for the treatment of patients with chronic lymphocytic leukemia (CLL) who have a chromosomal abnormality called 17p deletion and who have been treated with at least one prior therapy. Venetoclax is the first FDA-approved treatment that targets the B-cell lymphoma 2 (BCL-2) protein, which supports cancer cell growth and is overexpressed in many patients with CLL.
The efficacy of venetoclax was evaluated in a single-arm clinical trial of 106 patients with CLL who had a 17p deletion and who had received at least one prior therapy. The subjects received oral venetoclax every day, beginning with 20 mg and increasing over a five-week period to 400 mg. The results showed that 80% of the subjects experienced complete or partial remission.
Source: FDA, April 11, 2016
Descovy for HIV-1 Infection
Descovy (emtricitabine 200 mg/tenofovir alafenamide 25 mg, Gilead Sciences), a fixed-dose combination for the treatment of human immunodeficiency virus-1 (HIV-1) infection, has received FDA approval. Descovy is indicated for use in combination with other antiretroviral agents in adults and pediatric patients 12 years of age and older. Descovy is not indicated for use as pre-exposure prophylaxis to reduce the risk of sexually acquired HIV-1 infection in adults at high risk.
The label for Descovy includes a boxed warning regarding the risks of lactic acidosis or severe hepatomegaly with steatosis, and post-treatment acute exacerbation of hepatitis B.
Source: FDA, April 5, 2016
Defitelio for Liver Disease
The FDA has granted marketing approval for defibrotide sodium injection (Defitelio, Jazz Pharmaceuticals) for the treatment of adult and pediatric patients with hepatic veno-occlusive disease, also known as sinusoidal obstruction syndrome, with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).
In a phase 3 prospective study, the survival rate at day +100 after HSCT was 38% in 102 patients treated with defibrotide (6.25 mg/kg every six hours). In a phase 2 prospective study, the survival rate was 44% in 75 patients.
Source: Jazz Pharmaceuticals, March 30, 2016
Cinqair for Severe Asthma
Reslizumab (Cinqair, Teva Pharmaceuticals) has received FDA approval for use with other asthma medications for the maintenance treatment of severe asthma in patients 18 years of age and older. Reslizumab is approved for patients who have a history of severe asthma attacks (exacerbations) despite receiving their current asthma medications.
Reslizumab is a humanized interleukin-5 antagonist monoclonal antibody produced by recombinant DNA technology in murine myeloma nonsecreting 0 cells. The treatment reduces severe asthma attacks by reducing the levels of blood eosinophils, which contribute to the development of asthma.
Source: FDA, March 23, 2016
Taltz for Plaque Psoriasis
The FDA has approved ixekizumab injection 80 mg/mL (Taltz, Eli Lilly) for the treatment of moderate-to-severe plaque psoriasis in adult patients who are candidates for systemic therapy or phototherapy.
Ixekizumab is designed to specifically target interleukin-17A, a protein that plays a role in driving underlying inflammation in psoriasis. In three phase 3 studies, 87% to 90% of patients treated with ixekizumab demonstrated a significant improvement in their psoriasis plaques at 12 weeks.
Source: Eli Lilly, March 22, 2016
BromSite for Cataract Surgery Pain
The FDA has given the green light to BromSite (bromfenac ophthalmic solution, 0.075%, InSite Vision, Inc./Sun Pharma) for the treatment of postoperative inflammation and the prevention of ocular pain in patients undergoing cataract surgery.
BromSite is the first nonsteroidal anti-inflammatory drug approved by the FDA to prevent pain and to treat inflammation in the eye for patients undergoing cataract surgery. It is also the first bromfenac ophthalmic solution formulated in DuraSite, a polymer-based formulation that can be used to improve solubility, absorption, bioavailability, and residence time compared with that of conventional topical therapies, according to the manufacturer.
Source: Sun Pharma, April 9, 2016
Biosimilar and Generic Approvals
Inflectra for Multiple Indications
The FDA has approved infliximab-dyyb (Inflectra, Celltrion, Inc.) for multiple indications. The product is biosimilar to infliximab (Remicade, Janssen Biotech), which was originally licensed in 1998. This is the second time a biosimilar product has been cleared by the FDA. Infliximab-dyyb is indicated for the treatment of 1) adult and pediatric patients (6 years of age and older) with moderately to severely active Crohn’s disease who have shown an inadequate response to conventional therapy; 2) adult patients with moderately to severely active ulcerative colitis who have shown an inadequate response to conventional therapy; 3) patients with moderately to severely active rheumatoid arthritis in combination with methotrexate; 4) patients with active ankylosing spondylitis; 5) patients with active psoriatic arthritis; and 6) adult patients with chronic severe plaque psoriasis.
Source: FDA, April 5, 2016
Oxiconazole Nitrate Cream
Taro Pharmaceuticals USA has received FDA approval for oxiconazole nitrate cream, 1%, the first generic version of Oxistat cream (PharmaDerm). The topical antifungal is indicated for the treatment of tinea pedis, tinea cruris, and tinea corporis due to Trichophyton rubrum, T. mentagrophytes, or Epidermophyton occosum, and also for the treatment of tinea (pityriasis) versicolor due to Malassezia furfur.
Source: FDA, March 7, 2016, and Oxistat prescribing information
Final FDA approval has been granted to Teva Pharmaceuticals USA for sildenafil citrate tablets, the first generic version of Viagra (Pfizer). The erectile dysfunction drug will be available in 25-mg, 50-mg, and 100-mg strengths. Teva will begin marketing the product in 2017.
Source: FDA, March 9, 2016
Diclofenac Sodium Topical Gel
The FDA has approved the abbreviated new drug application for diclofenac sodium topical gel, 1%, a nonsteroidal anti-inflammatory drug indicated for the relief of osteoarthritis pain in the elbow, wrist, hand, knee, ankle, or foot. This first generic version of Voltaren gel (Novartis) is marketed by Amneal Pharmaceuticals.
Source: Amneal Pharmaceuticals, March 24, 2016
Mometasone Furoate Nasal Spray
Apotex Corp. has received FDA approval for the marketing of mometasone furoate nasal spray, 50 mcg. The product, a first-time generic of Nasonex (Merck), is a once-daily anti-allergen indicated for the treatment of nasal congestion.
Source: Apotex Corp., March 24, 2016
Bendamustine HCl for Injection
The FDA has granted approval to both Glenmark Pharmaceuticals, Ltd., and Innopharma Licensing LLC for bendamustine hydrochloride (HCl) for injection single-dose vials (25 mg/vial, 100 mg/ vial). The drug is used to treat chronic lymphocytic leukemia and indolent B-cell non-Hodgkin lymphoma that has progressed during or within six months of treatment with rituximab or a rituximab-containing regimen. The FDA initially approved bendamustine in 2008 under the brand name Treanda (Cephalon, Inc.).
Sources: FDA, March 24, 2016, and Treanda prescribing information
Daptomycin for Injection
Teva Pharmaceuticals USA has earned FDA approval for the lipopeptide antibacterial drug daptomycin for injection (500 mg/vial). Initially approved in 2003 as Cubicin (Merck), daptomycin is indicated for the treatment of complicated skin and skin structure infections and Staphylococcus aureus bloodstream infections (bacteremia).
Sources: FDA, March 24, 2016, and Cubicin prescribing information
Oxycodone Capsules, 5 mg
The FDA has approved the abbreviated new drug application for oxycodone hydrochloride capsules, 5 mg (ANI Pharmaceuticals), a Schedule II narcotic product. Oxycodone is an opioid agonist indicated for the management of moderate-to-severe acute and chronic pain where the use of an opioid analgesic is appropriate.
Source: ANI Pharmaceuticals, April 5, 2016
Polymyxin B for Injection
Aurobindo Pharma Ltd. has received final approval from the FDA to manufacture and market polymyxin B for injection USP, 500,000 units/vial. The product is expected to launch in the second quarter of fiscal year 2016–2017. The approved product is bioequivalent and therapeutically equivalent to polymyxin B for injection USP, 500,000 units/vial, marketed by Eurohealth International Sarl.
Source: Aurobindo Pharma, April 5, 2016
Zolpidem Sublingual Tablets
Lupin Pharmaceuticals has launched zolpidem sublingual tablets 1.75 mg and 3.5 mg, a generic equivalent of Intermezzo sublingual tablets (Purdue Pharma). The generic product is indicated for use as needed for the treatment of insomnia when a middle-of-the-night awakening is followed by difficulty returning to sleep.
Source: Lupin Pharmaceuticals, April 5, 2016
Tramadol Extended-Release Tablets
Mylan N.V. has announced the U.S. launch of tramadol hydrochloride extended-release tablets USP, 100 mg, 200 mg, and 300 mg—the generic version of Ultram extended-release tablets (Valeant). The product is indicated for the management of moderate to moderately severe chronic pain in adults who require around-the-clock treatment of their pain for an extended period.
Source: Mylan, March 28, 2016
Xalkori for ROS1-Positive Lung Cancer
The FDA has approved a supplemental new drug application for crizotinib (Xalkori, Pfizer) to treat patients with metastatic non–small-cell lung cancer (NSCLC) whose tumors are ROS1-positive. ROS1 rearrangements occur when the ROS1 gene attaches to another gene and changes the way each gene normally functions, which can contribute to cancer cell growth.
The FDA’s approval was based on results from a single-arm phase 1 trial in which 50 patients with ROS1-positive metastatic NSCLC were treated with oral crizotinib (250 mg twice daily). Crizotinib achieved an objective response rate of 66%. There was one complete response and 32 partial responses. The median duration of response was 18.3 months.
Crizotinib is also indicated for patients with metastatic NSCLC whose tumors are anaplastic lymphoma kinase-positive, as detected by an FDA-approved test.
Source: Pfizer, March 11, 2016
Evomela for Multiple Myeloma
The FDA has granted approval of melphalan (Evomela, Spectrum Pharmaceuticals) for two indications: 1) use as a high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation in patients with multiple myeloma (MM), and 2) palliative treatment of patients with MM for whom oral therapy is not appropriate. This is the first product to be FDA-approved for the high-dose conditioning indication in MM.
Evomela was approved by the FDA based on its bioequivalence to the standard melphalan formulation (Alkeran, GlaxoSmithKline) in a phase 2 clinical study. The Evomela formulation of melphalan does not contain propylene glycol.
Source: Spectrum Pharmaceuticals, March 15, 2016
FDA REVIEW ACTIVITIES
Priority Review Status
Atezolizumab for Lung Cancer And for Urothelial Carcinoma
The FDA has accepted a biologics license application and granted priority review designations to atezolizumab (Genentech) for the treatment of two groups of patients:
- Patients with locally advanced or metastatic non–small-cell lung cancer whose disease expresses programmed death ligand-1 (PD-L1) proteins, as determined by an FDA-approved test, and who have progressed on or after platinum-containing chemotherapy
- Patients with locally advanced or metastatic urothelial carcinoma who had disease progression during or after platinum-based chemotherapy in the metastatic setting, or whose disease worsened within 12 months of receiving platinum-based chemotherapy before surgery (neoadjuvant) or after surgery (adjuvant)
Atezolizumab is an investigational monoclonal antibody designed to bind directly with PD-L1 on tumor cells and tumor-infiltrating immune cells, thereby blocking interactions with PD-1 and B7.1 receptors. By inhibiting PD-L1, atezolizumab may enable the activation of T cells. Atezolizumab may also affect normal cells.
Source: Roche, March 15 and April 10, 2016
Orphan Drug Designations
VAL-083 for Medulloblastoma
The FDA has granted orphan drug status to VAL-083 (DelMar Pharmaceuticals), a small-molecule chemo therapeutic, for the treatment of patients with medullo-blastoma. The investigational drug candidate previously received an orphan designation for glioblastoma.
Medulloblastoma is the most common malignant pediatric brain tumor, accounting for 15% to 30% of all childhood intracranial neoplasms. Although multidisciplinary treatment has significantly improved the five-year survival rate in children, the prognosis for certain subtypes of medulloblastoma and for recurrent disease remains poor, with median overall survival of less than one year.
Source: DelMar Pharmaceuticals, March 15, 2016
PNT2258 for Lymphoma
The oncology drug candidate PNT2258 (ProNAi Therapeutics) has been granted an orphan drug designation by the FDA for the treatment of diffuse large B-cell lymphoma (DLBCL). PNT2258 is designed to target cancers that over-express BCL2, an important and validated oncogene known to be dysregulated in many types of cancer. BCL2 over-expression is thought to be a key driver of DLBCL, an aggressive form of cancer that is the most prevalent form of non-Hodgkin’s lymphoma.
Source: ProNAi Therapeutics, March 14, 2016
Advisory Committee Actions
Ocaliva for Liver Disease
The FDA’s Gastrointestinal Drugs Advisory Committee has voted 17 to 0 to recommend accelerated approval of Ocaliva (obeticholic acid, Intercept Pharma ceuticals) for the treatment of patients with primary biliary cirrhosis, recently renamed primary biliary cholan-gitis (PBC). The target date for the FDA to act under the Prescription Drug User Fee Act is May 29, 2016.
Approval is being sought for the treatment of PBC in patients with an inadequate response to, or who are unable to tolerate, ursodeoxycholic acid, the only approved therapy for PBC.
Source: Intercept Pharmaceuticals, April 7, 2016
KP201/APAP for Acute Pain
A joint meeting of the FDA’s Anesthetic and Analgesic Drug Products Advisory Committee and the agency’s Drug Safety and Risk Management Advisory Committee has been scheduled for May 5, 2016, to review the new drug application (NDA) for KP201/APAP (KemPharm, Inc.) for the short-term management of acute pain.
KP201/APAP is a combination of a pro-drug of hydrocodone (benzhydrocodone hydrochloride [KP201]) and acetaminophen (APAP) that is being developed as an abuse-deterrent immediate-release prodrug of hydrocodone bitartrate/ APAP. The NDA for KP201/APAP was accepted and granted priority review by the FDA in February 2016. The agency has set a target action date of June 9, 2016.
Source: KemPharm, March 14, 2016
Oral Relistor for Opioid-Induced Constipation
The FDA has extended the action date for its review of the new drug application for an oral formulation of methylnaltrex-one bromide (Relistor, Valeant Pharmaceuticals International) by three months to July 19, 2016. The FDA extended the date to allow a full review of new information submitted by Valeant at the agency’s request.
Methylnaltrexone bromide, administered as a subcutaneous injection, was approved in the U.S. in 2008 for the treatment of opioid-induced constipation in adults with chronic noncancer pain and in adults with advanced illness who are receiving palliative care. The drug is a mu-opioid receptor antagonist.
Source: Valeant, April 4, 2016
DRUG LABELING CHANGES
Saxagliptin and Alogliptin Linked With Heart Failure
An FDA safety review has found that type-2 diabetes medications containing saxagliptin or alogliptin may increase the risk of heart failure, particularly in patients who already have heart or kidney disease. As a result, the agency has added new warnings and precautions to the labels of medications that contain saxagliptin or alogliptin to underscore the potential increased risk of heart failure.
Saxagliptin and alogliptin are part of the class of dipeptidyl peptidase-4 inhibitor drugs, which are used with diet and exercise to lower blood sugar in adults with type-2 diabetes. Saxagliptin-containing medications include AstraZeneca’s Onglyza (saxagliptin) and Kombiglyze XR (extended-release saxagliptin/met-formin), and alogliptin-containing medications include Takeda’s Nesina (alogliptin), Kazano (alogliptin/metformin), and Oseni (alogliptin/pioglitazone).
Source: FDA, April 5, 2016
Stiolto Respimat QOL Data
The FDA has approved a supplemental new drug application for Stiolto Respimat (tiotropium bromide and olodaterol inhalation spray, Boehringer Ingelheim) that adds data to the product labeling showing improvement in health-related quality of life (hrQOL) among patients with chronic obstructive pulmonary disease. These data, which are from the OTEMTO 1 and 2 clinical studies, show a clinically meaningful improvement in hrQOL as measured by the St. George’s Respiratory Questionnaire, which is a disease-specific patient-reported instrument that evaluates symptoms, activities, and the impact of the disease on daily life.
Source: Boehringer Ingelheim, March 29, 2016
CLINICAL TRIAL NEWS
Ridinilazole for C. difficile Infection
Positive phase 2 results highlighting the potential of ridinilazole (Summit Therapeutics), a small-molecule antibiotic, in the treatment of patients with Clostridium difficile infection (CDI) were reported at the 26th European Congress of Clinical Microbiology and Infectious Diseases. The findings included a reduced recurrence rate and a statistically superior rate of sustained clinical response (SCR) in patients with CDI treated with ridinilazole compared with those receiving the standard of care (vancomycin).
The SCR rates for ridinilazole and vancomycin were 67% and 42%, respectively, and the recurrence rates were 14% and 35%. The cure rates at the end of treatment were 78% and 70% for the two drugs, respectively.
Source: Summit Therapeutics, April 11, 2016
Xeljanz for Psoriatic Arthritis
Positive results have been reported from a phase 3 study investigating tofacitinib (Xeljanz, Pfizer) for the treatment of patients with psoriatic arthritis (PsA). The study evaluated the efficacy and safety of tofacitinib 5 mg and 10 mg twice daily in adults with active PsA who had shown an inadequate response to at least one conventional synthetic disease-modifying antirheumatic drug and who were tumor necrosis factor inhibitor–naïve. The trial met its primary efficacy endpoints, demonstrating that both the 5-mg and 10-mg twice-daily regimens were superior to placebo at three months.
Tofacitinib is a Janus kinase (JAK) inhibitor. It is the only once-daily oral JAK inhibitor approved for the treatment of patients with moderate-to-severe rheumatoid arthritis.
Source: Pfizer, April 5, 2016
Benjorna for ADHD
Ironshore Pharmaceuticals and Development, Inc., reported positive clinical data from a phase 3 pivotal trial of its investigational drug product Benjorna (delayed-release and extended-release methylphenidate capsules) in 153 pediatric patients with attention-deficit/ hyperactivity disorder (ADHD). Patients treated with Benjorna demonstrated a statistically significant improvement compared with those given placebo (P = 0.01), based on a composite measure from 8
Source: Ironshore, April 5, 2016
Cimzia Versus Humira for RA
Top-line results have been reported from the first head-to-head superiority study of two treatments in the anti-tumor necrosis factor class, comparing certolizumab pegol (Cimzia, UCB) plus methotrexate (MTX) with adalimumab (Humira, AbbVie) plus MTX in adult patients with moderate-to-severe rheumatoid arthritis (RA) who are inadequate responders to MTX. The primary endpoints for superiority were not met, as the results between certolizumab and adalimumab were numerically comparable, showing that the percentage of patients achieving a 20% improvement at three months was 69.2% versus 71.4%, respectively, and that the percentage of patients achieving a state of low disease activity at two years was 35.5% versus 33.5%, respectively.
Source: UCB, March 24, 2016
Vyxeos for AML
Positive results have been reported from a phase 3 trial of cytarabine/daunorubicin liposome for injection (Vyxeos, Celator Pharmaceuticals) in patients with high-risk (secondary) acute myeloid leukemia compared with the standard-of-care regimen of cytarabine and daunorubicin known as 7+3. The trial met its primary endpoint, demonstrating a statistically significant improvement in overall survival.
The median overall survival for patients treated with cytarabine/daunorubicin was 9.56 months compared with 5.95 months for patients receiving 7+3, representing a 3.61-month improvement in favor of the liposome treatment (P = 0.005). The pro portion of patients alive at 12 months after randomization was 42% in the cytarabine/ daunorubicin arm compared with 28% in the 7+3 arm.
Source: Celator Pharmaceuticals, March 14, 2016
FG-3019 for Pulmonary Fibrosis
The European Respiratory Journal has published the results from a phase 2 study in subjects with idiopathic pulmonary fibrosis (IPF) treated for 48 weeks with the investigational drug FG-3019 (FibroGen, Inc.), a monoclonal antibody that inhibits the activity of connective tissue growth factor, a central mediator of fibrotic disease. A total of 89 subjects received one or more doses of FG-3019 (either 15 mg/kg or 30 mg/kg) every three weeks. Of these participants, 75 completed 24 weeks of treatment and 66 completed 48 weeks.
While most of the subjects in the study (65%) showed an increase in fibrosis, 35% experienced stable or improved fibrosis at week 48, as measured by high-resolution computed tomography. At both 24 and 48 weeks, improvements in lung fibrosis correlated with improvements in lung function.
Source: FibroGen, March 11, 2016
Encenicline Fails in Schizophrenia Study
Forum Pharmaceuticals announced top-line results from two phase 3 clinical trials in patients with cognitive impairment in schizophrenia. While encenicline demonstrated favorable safety and tolerability profiles in both studies, neither trial met its coprimary endpoints of the effect on cognitive function and patient function. Encenicline is an orally administered selective agonist of the alpha 7 receptor found on hippocampal and cortical neurons involved in cognition.
Source: Forum Pharmaceuticals, March 25, 2016
Brilinta Misses in Stroke Trial
AstraZeneca has announced the results from the pivotal SOCRATES trial, which assessed the efficacy of ticagrelor (Brilinta) 90 mg twice daily compared with aspirin 100 mg once daily in patients with acute ischemic stroke or transient ischemic attack. The primary efficacy endpoint of the time to the first occurrence of any event from the composite of stroke (ischemic or hemorrhagic), myocardial infarction, and death was not met. Fewer vascular events were observed in the ticagrelor arm compared with the aspirin arm in the overall trial population, but the trend did not reach statistical significance.
Ticagrelor is a direct-acting platelet P2Y12 receptor antagonist in a chemical class called cyclo-pentyl-triazolo-pyrimidines. Ticagrelor works by inhibiting platelet activation.
Source: AstraZeneca, March 23, 2016
Lilly Changes Phase 3 Endpoint
Eli Lilly has announced a change to the primary endpoint for the phase 3 EXPEDITION3 trial of solanezumab, a humanized monoclonal immunoglobulin G1 antibody, in subjects with mild Alzheimer’s dementia. The original study design included coprimary endpoints of cognition and function. Emerging scientific evidence supports the idea that cognitive decline precedes and predicts functional decline in Alzheimer’s disease, particularly in earlier stages of the disease. Therefore, Lilly decided to amend the EXPEDITION3 trial to include a single primary endpoint of cognition. Functional outcomes will be measured during the trial in the same manner as previously designed, using both the Alzheimer’s Disease Cooperative Study– Instrumental Activities of Daily Living and the Functional Assessment Questionnaire. These two functional outcomes will now be considered key secondary endpoints for the EXPEDITION3 study.
Source: Eli Lilly, March 15, 2016
Epidiolex for Seizures
GW Pharmaceuticals has initiated a phase 3 dose-ranging study of Epidiolex (cannabidiol) as an adjunctive therapy for seizures associated with tuberous sclerosis complex (TSC), a rare genetic disorder that causes nonmalignant tumors to form in many different organs, particularly the brain and skin. The most common symptom of TSC is epilepsy.
Epidiolex is a liquid formulation of plant-derived cannabidiol that is in development for the treatment of several rare pediatric epilepsy disorders.
The 16-week dose-ranging trial will compare Epidiolex and placebo in approximately 200 patients, ages 1 to 65 years, to determine the safety and efficacy of Epidiolex as an adjunctive antiepileptic treatment. The trial’s primary endpoint is the percentage change from baseline in seizure frequency during the treatment period.
Source: GW Pharmaceuticals, April 11, 2016
AZD3293 for Early Alzheimer’s
Eli Lilly and AstraZeneca have announced that the phase 2/3 AMARANTH study of AZD3293, an oral beta secretase-cleaving enzyme (BACE) inhibitor currently in development as a potential treatment for early Alzheimer’s disease (AD), will continue to phase 3.
In phase 1 studies, AZD3293 reduced the levels of amyloid beta in the cerebrospinal fluid of subjects with AD and in healthy volunteers. The progression of AD is characterized by the accumulation of amyloid plaque in the brain. BACE is an enzyme associated with the development of amyloid beta. The inhibition of BACE is expected to prevent the formation of amyloid plaque and eventually to slow the progression of the disease.
Source: Eli Lilly, April 8, 2016
Durvalumab/Motolimod Combo for Ovarian Cancer
A phase 1/2, open-label, multi national trial has been launched to evaluate a combination of the investigational antibody durvalumab (MedImmune), a programmed death ligand-1 (PD-L1) inhibitor, and the investigational toll-like receptor 8 (TLR8) agonist motolimod (VentiRx) added to chemotherapy in patients with locally advanced or recurrent ovarian cancer that has become resistant to platinum chemotherapy.
The researchers expect that the activation of TLR8 by motolimod will create conditions within tumors that enhance the effects of durvalumab. The trial is being conducted by the Ludwig Institute for Cancer Research and the Cancer Research Institute, both located in New York.
Source: Ludwig Institute for Cancer Research, April 8, 2016
Avelumab and Inlyta for Renal Cell Carcinoma
Merck and Pfizer have announced the treatment of the first patient in a phase 3 study of avelumab, an investigational fully human anti-programmed death ligand-1 (anti–PD-L1) immunoglobulin G1 monoclonal antibody, in patients with renal cell carcinoma (RCC). The study, JAVELIN Renal 101, is the first pivotal trial investigating avelumab in combination with Inlyta (axitinib), a tyrosine kinase inhibitor (TKI), in patients with previously untreated advanced RCC, and is the only phase 3 trial currently evaluating an anti–PD-L1 immunotherapy in combination with a vascular endothelial growth factor-receptor TKI in this setting.
Source: Merck, April 5, 2016
Relamorelin for Diabetic Gastroparesis
Motus Therapeutics has completed enrollment in a phase 2b trial assessing the efficacy and safety of relamorelin (RM-131), a ghrelin agonist, for the treatment of gastroparesis in patients with type-1 and type-2 diabetes. Ghrelin is a peptide hormone produced in the stomach that stimulates gastrointestinal motility.
The randomized, double-blind, placebo-controlled study is evaluating the safety and efficacy of dosing regimens ranging from 10 to 100 mcg administered twice daily over three months. The trial enrolled 396 patients with diabetic gastroparesis at clinical sites in the U.S and Europe.
Source: Motus Therapeutics, April 4, 2016
Sublingual Sufentanil For ER Pain
AcelRx Pharmaceuticals has initiated the extension phase of a phase 3, open-label study of sublingual sufentanil (ARX-04) for the treatment of adults who present to the emergency room (ER) with moderate-to-severe acute pain associated with trauma or injury. This ongoing study has completed its initial phase, enrolling 40 patients who each received a single dose of sublingual sufentanil. In the extension phase, up to an additional 100 patients may be enrolled, each of whom may receive multiple doses of sub-lingual sufentanil, given hourly as needed for pain, for up to four doses. The trial’s primary endpoint is the time-weighted summed pain intensity difference to baseline over one hour.
ARX-04 is a noninvasive investigational product candidate consisting of 30-mcg sufentanil tablets delivered sublingually via a disposable, prefilled, single-dose applicator.
Source: AcelRx Pharmaceuticals, March 14, 2016
RP-G28 for Lactose Intolerance
Ritter Pharmaceuticals has announced the initiation of a phase 2b/3 clinical trial of RP-G28, which has the potential to be the first FDA-approved treatment for lactose intolerance. The double-blind, placebo-controlled, three-arm study is enrolling approximately 350 patients with symptoms of lactose intolerance. The patients will undergo a 30-day treatment process, followed by a 30-day post-treatment evaluation. In addition, the study will evaluate the participants’ microbiomes, expanding the knowledge of the effects that RP-G28 may have on positively adapting and shifting the gut microbiota for health benefits. Top-line results are expected in early 2017.
Source: Ritter Pharmaceuticals, March 14, 2016
Clivatuzumab Tetraxetan Study In Pancreatic Cancer Ended
Immunomedics, Inc., has terminated its phase 3 PANCRIT-1 trial of yttrium-90-labeled (90Y) clivatuzumab tetraxetan in patients with metastatic pancreatic cancer who had received at least two prior therapies, one of which must have been a gemcitabine-containing regimen.
The decision to end the trial early was based on a recommendation from an independent data and safety-monitoring board after more than 50% of the required 371 deaths had occurred. The board’s interim analysis showed that the treatment arm of 90Y-clivatuzumab tetraxetan combined with low-dose gemcitabine and best supportive care did not demonstrate a sufficient improvement in overall survival compared with placebo plus low-dose gemcitabine and best supportive care.
Source: Immunomedics, March 14, 2016
First Wireless Pacemaker
The FDA has approved the first pacemaker that does not require the use of wired leads to provide an electrical connection between the pulse-generating device and the heart. While the Micra Transcatheter Pacing System (Medtronic) works like other pacemakers to regulate heart rate, the self-contained, inch-long device is implanted directly in the right ventricle chamber of the heart.
The FDA evaluated data from a clinical trial of 719 patients implanted with the Micra device. In that trial, 98% of the patients had adequate heart pacing (known as the pacing capture threshold) six months after the device was implanted. Complications occurred in fewer than 7% of participants and included prolonged hospitalizations, deep vein thrombosis, pulmonary embolism, heart injury, device dislocation, and heart attacks.
Source: FDA, April 6, 2016
HeartLight Endoscopic Ablation System
CardioFocus, Inc., has received pre-market approval from the FDA for its HeartLight Endoscopic Ablation System for the treatment of patients with paroxysmal atrial fibrillation (AF). The approved submission contained safety and efficacy data from the company’s HeartLight U.S. Pivotal Clinical Study, a randomized, controlled trial in which 353 participants were randomized at 19 arrhythmia centers in the United States.
The trial results showed that when a single ablation procedure using the HeartLight system was performed, most patients experienced freedom from paroxysmal AF at 12 months. In addition, the primary safety and efficacy endpoints were met.
Source: CardioFocus, April 4, 2016
TX2 MicroTip for Chronic Tendinosis
Tenex Health Inc. has obtained FDA 510(k) clearance for its new TX2 Micro-Tip. The device will be used primarily to address tendinosis of the shoulder and hip, two areas that were more difficult to reach using the original TX1 MicroTip because of its length. The TX2 MicroTip is a two-inch-long disposable surgical instrument that uses ultrasonic energy to specifically cut and remove targeted soft tissue.
Source: Tenex Health, March 29, 2016
Concurrent Use of Wound Management Devices
ACell, Inc., has received additional FDA approval for its MicroMatrix device, which describes the concurrent use of MicroMatrix in conjunction with Cytal Wound Matrix or Cytal Burn Matrix devices. MicroMatrix facilitates coverage of the wound bed, especially in irregular wounds. Cytal Wound Matrix (one-layer, two-layer, three-layer, and six-layer) and Cytal Burn Matrix provide a scaffold for cell infiltration and host tissue deposition.
Source: ACell, March 23, 2016
The FDA has granted 510(k) clearance to CardiacAssist, Inc., for its new TandemLung oxygenator, a medical device that acts as an artificial lung to infuse oxygen and remove carbon dioxide from the blood. The device is paired with the TandemHeart blood pump.
The TandemLung device uses a radial flow design coupled with polymethylpentene fibers to transfer oxygen into the blood for patients requiring cardiac or respiratory support. It is intended for use in adult patients for extracorporeal circulation during cardiopulmonary bypass for up to six hours.
Source: CardiacAssist, March 15, 2016
OTHER DEVICE NEWS
Spirulina Breath Test For Gastroparesis
Cairn Diagnostics has launched the 13C-Spirulina gastric emptying breath test (GEBT). The GEBT is intended for measurements of the rate of solid-phase gastric emptying and as an aid in the diagnosis of gastroparesis in symptomatic adults. The test was approved by the FDA in April 2015. The GEBT is nonradioactive and noninvasive and can be administered at a physician’s office, at a laboratory collection center, or in a tertiary care setting.
Source: Cairn Diagnostics, March 29, 2016
ACT Ablation Technology
Advanced Cardiac Therapeutics (ACT), developer of DiamondTemp, a proprietary open-irrigated, temperature-controlled, radio frequency (RF) ablation technology, has completed enrollment in its first-inhuman clinical study of 35 patients with paroxysmal atrial fibrillation. The study is designed to evaluate the safety and effectiveness of the ACT system, which includes the DiamondTemp catheter, RF generator, irrigation pump, and various accessories.
Source: ACT, March 28, 2016
Cyborg Heart Patch
The Cyborg cardiac patch, developed at Tel Aviv University, combines organic and engineered parts. The patch can contract and expand like human heart tissue but regulates itself like a machine, according to Professor Tal Dvir and his team.
The researchers first developed thick bionic tissue suitable for transplantation. This engineered tissue featured electronics that could sense tissue function and provide electrical stimulation. In addition, electroactive polymers were integrated with the electronics. On activation, these polymers could release medications, such as growth factors or small molecules, on demand.
“Imagine that a patient is just sitting at home, not feeling well,” Dr. Dvir said. “His physician will be able to log onto his computer and this patient’s file—in real time. He can view data sent remotely from sensors embedded in the engineered tissue and assess exactly how his patient is doing. He can intervene to properly pace the heart and activate drugs to regenerate tissue from afar.”
Source: Tel Aviv University, March 14, 2016