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P T. 2016;41(4): 208-210, 212-214, 218-219

Drug and Device News April 2016

NEW DRUG APPROVALS

Idelvion for Hemophilia B

The FDA has approved Idelvion (coagulation factor IX [recombinant], albumin fusion protein, CSL Behring), a long-acting albumin fusion protein linking recombinant coagulation factor IX with recombinant albumin for the treatment of patients with hemophilia B. Idelvion is the first factor IX therapy that delivers protection with up to 14-day dosing in appropriate patients, according to CSL Behring. This dosing interval is achieved while maintaining high levels of factor activity, thereby reducing the monthly number of units needed for prophylaxis therapy.

Idelvion is a recombinant protein that temporarily replaces the missing coagulation factor IX needed for effective hemostasis. It is indicated for children and adults with hemophilia B for routine prophylaxis to prevent or reduce the frequency of bleeding episodes; for on-demand control and prevention of bleeding episodes; and for the perioperative management of bleeding (around the time of surgery).

Source: CSL Behring, March 4, 2016

Odefsey for HIV Infection

Odefsey (emtricitabine 200 mg/rilpivirine 25 mg/tenofovir alafenamide 25 mg) has been approved by the FDA for the treatment of human immunodeficiency virus-1 (HIV-1) infection in certain patients. Emtricitabine and tenofovir alafenamide (TAF) are from Gilead Sciences, and rilpivirine is from Janssen Sciences Ireland. Odefsey is Gilead’s second regimen based on TAF, a tenofovir prodrug, to receive FDA approval.

Odefsey is indicated as a complete regimen for the treatment of HIV-1 infection in patients 12 years of age and older who have no antiretroviral treatment history and HIV-1 RNA levels less than or equal to 100,000 copies per mL. Odefsey is also indicated as a replacement for a stable antiretroviral regimen in patients who are virologically suppressed (HIV-1 RNA less than 50 copies per mL) for at least six months, with no history of treatment failure and no known substitutions associated with resistance to the individual components of Odefsey.

Source: Gilead Sciences, March 1, 2016

Aczone for Acne

Allergan has received approval from the FDA to market dapsone gel, 7.5% (Aczone), a topical sulfone for the treatment of acne vulgaris in patients 12 years of age and older. The product may be used to treat both inflammatory and noninflammatory acne in a once-daily application.

The safety and efficacy of dapsone gel, 7.5%, were evaluated in two identically designed 12-week studies involving a total of 4,340 acne patients. Dapsone gel reduced inflammatory lesions by 15.8 lesions (55%) compared with 13.9 lesions (48%) with vehicle, and noninflammatory lesions were reduced by 20.7 lesions (45%) compared with 18.0 lesions (39%), respectively. The Global Acne Assessment Score success rate in patients treated with dapsone was 30% compared with 21% with vehicle.

Source: Allergan, February 25, 2016

Briviact for Partial-Onset Seizures

The FDA has approved brivaracetam (Briviact, UCB, Inc.), a high-affinity synaptic vesicle protein 2A (SV2A) ligand, as an add-on treatment to other medications to treat partial-onset seizures in patients 16 years of age and older with epilepsy.

The efficacy of brivaracetam was studied in three clinical trials involving 1,550 subjects. The medication was effective in reducing the frequency of seizures. The most common adverse events included drowsiness, dizziness, fatigue, nausea, and vomiting.

Brivaracetam must be dispensed with a medication guide for patients, which provides important information about the medication’s use and risks. As is true for all drugs that treat epilepsy, the most serious risks include thoughts about suicide, attempts to commit suicide, feelings of agitation, new or worsening depression, aggression, and panic attacks.

Source: FDA, February 19, 2016

Generic Approvals

Sumatriptan Nasal Spray

The FDA has approved an abbreviated new drug application for sumatriptan nasal spray USP, 5 mg/spray and 20 mg/spray, the first therapeutic equivalent of Imitrex nasal spray (GlaxoSmith-Kline). The generic version is marketed by Lannett Company, Inc.

Sources: FDA, February 19, 2016; Lannett Company, Inc., February 22, 2016

Almotriptan Malate Tablets

Ajanta Pharma, based in India, has received final approval from the FDA for almotriptan malate tablets (6.25 mg and 12.5 mg), a generic version of Axert (Janssen), for the treatment of acute migraine pain.

Source: Ajanta Pharma, March 7, 2016

Generic Adderall XL

The FDA has approved the abbreviated new drug application for dextroamphetamine saccharate, amphetamine aspartate monohydrate, dextroamphetamine sulfate, and amphetamine sulfate (mixed salts of a single-entity amphetamine product) extended-release capsules, CII, 5 mg, 10 mg, 15 mg, 20 mg, 25 mg, and 30 mg. This generic version of Adderall XL (Shire) is marketed by Impax Laboratories. The product, a central nervous system stimulant, is indicated for the treatment of patients with attention-deficit/hyperactivity disorder.

Source: Impax, February 17, 2016

Temozolomide Capsules

Kremers Urban Pharmaceuticals, Inc., has received FDA approval of its abbreviated new drug application for temozolomide capsules 5 mg, 20 mg, 100 mg, 140 mg, 180 mg, and 250 mg––the therapeutic equivalent to the reference-listed drug Temodar capsules (Merck). In 2015, U.S. sales of temozolomide totaled approximately $206 million.

Source: Lannett Company, Inc., February 12, 2016

NEW INDICATIONS

Faslodex, Ibrance Combination For Metastatic Breast Cancer

The FDA has expanded the indications of fulvestrant (Faslodex, Astra Zeneca) and palbociclib (Ibrance, Pfizer) to approve their use in combination for women with hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2–) advanced or metastatic breast cancer whose disease has progressed after endocrine therapy.

In the phase 3 PALOMA-3 trial, the combination of fulvestrant 500 mg and palbociclib 125 mg resulted in a 4.9-month improvement in progression-free survival (PFS) over fulvestrant and placebo in women with HR+/HER2– advanced or metastatic breast cancer whose disease had progressed after endocrine therapy. Improvements in PFS were seen irrespective of the patients’ menopausal status.

Fulvestrant was originally approved in 2002 as monotherapy for postmenopausal women with HR+ metastatic breast cancer whose disease had progressed after antiestrogen therapy.

Palbociclib was first approved in February 2015, and is also indicated for the treatment of HR+, HER2– advanced or metastatic breast cancer in combination with letrozole as initial endocrine-based therapy in postmenopausal women. The indication in combination with letrozole was approved based on PFS. Palbociclib is the only cyclin-dependent kinase 4/6 inhibitor approved by the FDA.

Sources: Pfizer, February 19, 2016, and AstraZeneca, March 3, 2016

Afinitor for NET Of Lung Origin

The FDA has approved everolimus (Afinitor, Novartis) for the treatment of adults with progressive, well-differentiated, nonfunctional neuroendocrine tumors (NETs) of gastrointestinal or lung origin that are unresectable, locally advanced, or metastatic. It is the first treatment approved for progressive, nonfunctional NETs of lung origin.

Afinitor is now approved in the U.S. for use in the three most common types of advanced NETs (stomach, intestine, and lung).

Source: Novartis, March 1, 2016

Harvoni for HCV Infections

The FDA has approved additional indications for ledipasvir/sofosbuvir (Harvoni, Gilead Sciences) for use in chronic hepatitis C virus (HCV) patients with advanced liver disease. Harvoni in combination with ribavirin for 12 weeks was approved for use in chronic HCV genotype 1- or 4-infected liver transplant recipients without cirrhosis or with compensated cirrhosis (Child–Pugh A), and for HCV genotype 1-infected patients with decompensated cirrhosis (Child–Pugh B or C), including those who have undergone liver transplantation. Harvoni is now approved for use in a broader range of patient populations, including HCV genotypes 1, 4, 5, and 6, HCV/HIV-1 coinfection, HCV genotype 1 and 4 liver transplant recipients, and genotype 1-infected patients with decompensated cirrhosis.

Source: Gilead Sciences, February 16, 2016

NEW FORMULATION

Extended-Release Tofacitinib for RA

The FDA has given the green light to extended-release tofacitinib citrate tablets (Xeljanz XR, Pfizer) for the once-daily treatment of moderate-to-severe rheumatoid arthritis (RA) in patients who have had an inadequate response to or are intolerant of methotrexate. Extended-release tofacitinib is the first and only once-daily oral RA treatment in the class of Janus kinase (JAK) inhibitors.

Both the standard and extended-release formulations of tofacitinib (Xeljanz and Xeljanz XR) do not require injections or infusions. Either drug can be taken with or without methotrexate.

Tofacitinib is the only JAK inhibitor included in the 2015 American College of Rheumatology Guideline for the Treatment of Rheumatoid Arthritis.

Source: Pfizer, February 24, 2016

FDA REVIEW ACTIVITIES

Breakthrough Therapy Status

Oliceridine for Acute Pain

The FDA has granted breakthrough therapy status to intravenous oliceridine (TRV130) for the management of moderate-to-severe acute pain. After two successful phase 2 studies, oliceridine is now in phase 3 development. The ATHENA-1 safety and tolerability study is ongoing in approximately 900 patients, with pivotal studies expected to begin in the second quarter of 2016.

According to the drug’s developer (Trevena, Inc.), phase 2 study data for oliceridine showed “encouraging differentiation” from morphine.

Oliceridine was previously granted a fast-track designation by the FDA. It is the first mu receptor G protein pathway-selective modulator (mGPS)––a biased mu opioid receptor ligand that in preclinical studies activated pathways associated with analgesia while avoiding pathways that can promote respiratory depression and gastrointestinal dysfunction and limit analgesia.

Source: Trevena, Inc., February 22, 2016

Midostaurin for AML

Midostaurin (PKC412, Novartis) has received an FDA breakthrough therapy designation to for the treatment of adults with newly diagnosed acute myeloid leukemia (AML) who are FMS-like tyrosine kinase-3 (FLT3) mutation-positive, as detected by an FDA-approved test, and who are eligible to receive standard induction and consolidation chemotherapy.

The breakthrough therapy designation for midostaurin was primarily based on positive results from the phase 3 RATIFY trial. In that study, patients who received midostaurin and standard induction and consolidation chemotherapy experienced a significant improvement in overall survival (hazard ratio, 0.77; P = 0.0074) compared with those who received standard induction and consolidation chemotherapy alone. The median overall survival for patients in the midostaurin group was 74.7 months compared with 25.6 months for those in the chemotherapy group.

Source: Novartis, February 19, 2016

Ocrevus for Multiple Sclerosis

The FDA has granted a breakthrough therapy designation for ocrelizumab (Ocrevus, Genentech) for the treatment of patients with primary progressive multiple sclerosis (PPMS). There are currently no approved treatments for PPMS, a debilitating form of MS characterized by steadily worsening symptoms and typically without distinct relapses or periods of remission.

Ocrelizumab is an investigational humanized monoclonal antibody designed to selectively target CD20-positive B cells. These immune cells are thought to be a key contributor to myelin (nerve cell insulation and support) and axonal (nerve cell) damage, which can result in disability in patients with MS.

Source: Genentech, February 17, 2016

Durvalumab for Bladder Cancer

Durvalumab (MEDI4736, Astra-Zeneca), a human monoclonal antibody, has won FDA breakthrough therapy status for the treatment of patients with programmed death ligand-1 (PD-L1)– positive inoperable or metastatic urothelial bladder cancer whose tumor has progressed during or after one standard platinum-based regimen.

Durvalumab is being investigated as monotherapy or in combination with tremelimumab (Pfizer/AstraZeneca) in patients with non–small-cell lung cancer; head and neck, bladder, gastric, pancreatic, and blood cancers; and hepatocellular carcinoma. Durvalumab is also being tested in first-line bladder cancer as monotherapy and in combination with tremelimumab as part of the ongoing phase 3 DANUBE trial.

Source: AstraZeneca, February 17, 2016

Orphan Drug Designations

Riluzole for Spinocerebellar Ataxia

The FDA has granted orphan drug status to BHV-0223 (riluzole, Portage Biotech/Biohaven Pharmaceutical Holding Company), a glutamate-modulating agent, for the treatment of spinocerebellar ataxia, which is characterized by progressive ataxia attributed to various etiologies.

Ataxia is the loss of control of voluntary body movements and can involve unsteady gait, speech difficulties, and clumsiness, potentially progressing to the stage of difficulty with swallowing and breathing due to degenerative changes in the brain and spinal cord. Treatment is supportive, and no medications are currently approved for this potentially debilitating condition.

BHV-0223 is a formulation of riluzole that uses Zydis ODT fast-dissolve technology.

Source: Portage Biotech, March 3, 2016

FLAG-003 for Glioma

FLAG-003 (Flag Therapeutics) has secured an FDA orphan drug designation for the treatment of glioma. Gliomas, including glioblastoma, are the most aggressive forms of brain cancer and carry a poor prognosis for survival.

FLAG-003 is a small molecule that exerts both cytotoxic and cytostatic activity resulting from two distinct mechanisms of action. It possesses cytotoxic anti-tubulin activity by binding to the colchicine site of tubulin, thereby causing microtubule depolymerization. It also possesses antiangiogenic activity through binding and inhibition of receptor tyrosine kinase activity.

Source: Flag Therapeutics, March 1, 2016

TXA127 for Muscular Dystrophy

The FDA has granted orphan drug status to TXA127 (Tarix Orphan LLC) for the treatment of laminin-deficient congenital muscular dystrophy (LAMA2 MD or MDC1A). TXA127 is a pharmaceutical-grade formulation of the naturally occurring peptide angiotensin (1–7). The compound is part of the alternative renin–angiotensin system (RAS) and counteracts the classical RAS, which promotes hypertension, fibrosis, hypertrophy, and inflammation.

TXA127 previously received orphan drug designations for Duchenne’s muscular dystrophy and limb girdle muscular dystrophy.

Source: Tarix Orphan LLC, February 17, 2016

Cirara for CNS Indications

The FDA has granted an orphan drug designation to Cirara (Remedy Pharmaceuticals) for the treatment of acute subarachnoid hemorrhage (SAH) and acute spinal cord injury. SAH is a life-threatening form of stroke characterized by increased pressure on the brain due to blood and fluid buildup in the subarachnoid space.

Cirara is a high-affinity inhibitor of Sur1-Trpm4 channels, which are upregulated in ischemic and traumatic events. The compound is designed to close these channels and thus prevent or reduce edema in a variety of central nervous system–related indications. A phase 2 pilot study evaluating Cirara as a treatment for SAH patients is planned to begin in the second half of 2016.

Source: Remedy Pharmaceuticals, February 16, 2016

Scenesse for Cutaneous Porphyrias

Afamelanotide (Scenesse, Clinuvel Pharmaceuticals) has received an orphan drug designation from the FDA for the treatment of cutaneous variants of porphyria. The designation was awarded in recognition of the drug’s potential to treat or prevent symptoms in rare forms of porphyria. Afamelanotide was previously granted orphan status for the treatment of erythropoietic protoporphyria (EPP) and congenital erythropoietic porphyria (CEP).

Afamelanotide has been clinically evaluated as a photoprotective drug in patients with EPP, for which it received marketing authorization in Europe. It has been proposed that the treatment may also have a photoprotective effect in patients with other rare forms of cutaneous porphyria, such as CEP, variegate porphyria, and hereditary coproporphyria.

Source: Clinuvel Pharmaceuticals, February 12, 2016

ONL1204 for Retinal Detachment

The FDA has granted orphan drug status to ONL1204 (ONL Therapeutics) for the treatment of retinal detachment. ONL1204 is a first-in-class small-molecule peptide designed to protect key retinal cells, including photoreceptors, from cell death that occurs in a range of retinal diseases and conditions. Death of these retinal cells is the root cause of vision loss and the leading cause of blindness. ONL1204 will be delivered to patients upon diagnosis and is intended to block photoreceptor cells from dying until surgery can be completed.

ONL expects to advance ONL1204 into clinical trials for patients with retinal detachment in 2016.

Source: ONL Therapeutics, February 11, 2016

Advisory Committee Action

Remicade Biosimilar

The FDA’s Arthritis Advisory Committee has recommended approval of all eligible indications for CT-P13 (Celltrion/Pfizer), a proposed biosimilar to Janssen Biotech’s Remicade (infliximab), by a vote of 21 to 3. CT-P13 is the first biosimilar monoclonal antibody medication to be considered by the FDA. Korea-based Celltrion is seeking approval of CT-P13 for the treatment of a variety of autoimmune diseases, including Crohn’s disease, ulcerative colitis, rheumatoid arthritis (RA), and ankylosing spondylitis (AS).

The FDA panel based its recommendation on clinical data comparing CT-P13 with the U.S. reference product Remicade. The committee concluded that CT-P13 is highly similar to Remicade, notwithstanding minor differences in clinically inactive components. Further, the committee noted that there were no clinically meaningful differences between CT-P13 and Remicade in terms of the safety, purity, and potency of the product in the studied indications of RA and AS.

The FDA’s approval decision is expected in April 2016.

Source: Celltrion, February 10, 2016

New Applications

Enbrel for Pediatric Psoriasis

The FDA has accepted for review a supplemental biologics license application for the expanded use of etanercept (Enbrel, Amgen) to treat pediatric patients with chronic severe plaque psoriasis. If approved, etanercept would be the first systemic drug with this indication in the U.S. The FDA has set a Prescription Drug User Fee Act target action date of November 5, 2016.

Etanercept is a soluble form of a tumor necrosis factor receptor. It was first approved in 1998 for patients with moderate-to-severe rheumatoid arthritis. Etanercept was approved in 1999 to treat moderate-to-severe polyarticular juvenile idiopathic arthritis; in 2002 to treat psoriatic arthritis; in 2003 to treat patients with ankylosing spondylitis; and in 2004 to treat moderate-to-severe plaque psoriasis in adults.

Source: Amgen, March 10, 2016

Teflaro for Children With Skin Infections

The FDA has accepted for filing a supplemental new drug application for ceftaroline fosamil (Teflaro, Allergan). If approved, this filing will expand the label of ceftaroline beyond adults to include the treatment of children two months of age and older with acute bacterial skin and skin-structure infections, including infections caused by methicillin-resistant Staphylococcus aureus and community-acquired bacterial pneumonia caused by S. pneumoniae and other designated susceptible bacteria. Ceftaroline is a bactericidal cephalosporin with activity against both gram-positive and gram-negative pathogens.

Source: Allergan, February 16, 2016

CLINICAL TRIAL NEWS

Nucala for Asthma

New safety and efficacy data have shown that the risk–benefit profile generated through pivotal studies of the asthma treatment mepolizumab (Nucala, GlaxoSmithKline) was maintained over 52 weeks. Mepolizumab is a monoclonal antibody that binds to interleukin-5 (IL-5), preventing it from binding to its receptor on the surface of eosinophils. Inhibiting IL-5 binding in this way reduces blood, tissue, and sputum eosinophil levels.

A total of 651 asthma patients participated in the open-label COSMOS trial. The most frequently reported adverse events included nasal congestion (30%), upper respiratory tract infection (16%), asthma exacerbation (14%), and headache (14%). Treatment resulted in reductions in oral corticosteroid doses, which were accompanied by consistent reductions in the exacerbation rate and improvements in symptom control.

Mepolizumab is approved as an addon maintenance treatment for patients 12 years of age and older with severe asthma and an eosinophilic phenotype. It is not approved for the treatment of other eosinophilic conditions or for the relief of acute bronchospasm or status asthmaticus.

Source: GlaxoSmithKline, March 5, 2016

Victoza for Diabetes Patients

Novo Nordisk has announced the results of the LEADER trial, which investigated the cardiovascular safety of liraglutide (Victoza) during a period of up to five years in more than 9,000 adults with type-2 diabetes at high risk for major adverse cardiovascular events. The study’s primary endpoint was the first occurrence of a composite cardiovascular outcome comprising cardiovascular death, nonfatal myocardial infarction, or nonfatal stroke.

The trial met its primary endpoint, demonstrating a statistically significant reduction in cardiovascular risk with liraglutide. The superior reduction of major adverse cardiovascular events was derived from all three components of the endpoint.

Liraglutide is a human glucagon-like peptide-1 (GLP-1) analogue with an amino acid sequence that is 97% similar to that of endogenous human GLP-1. It was approved by the FDA in 2010 as an adjunct to diet and exercise to improve glycemic control in adults with type-2 diabetes.

Source: Novo Nordisk, March 4, 2016

Cabotegravir/Rilpivirine for HIV

Positive results have been reported from an ongoing phase 2b, parallel-group, open-label study investigating long- acting, injectable formulations of cabotegravir (ViiV Healthcare) and rilpivirine (Edurant, Janssen Sciences Ireland) as a two-drug treatment for patients with human immunodeficiency virus-1 (HIV-1) infection. The patients had already achieved HIV viral suppression with a three-drug oral regimen of cabotegravir plus two nucleoside reverse transcriptase inhibitors (NRTIs).

A total of 309 patients were assigned to one of three study arms during the maintenance period: an intramuscular (IM) cabotegravir long-acting formulation (400 mg) plus a rilpivirine long-acting formulation (600 mg) every four weeks; an IM cabotegravir long-acting formulation (600 mg) plus a rilpivirine long-acting formulation (900 mg) every eight weeks; or oral cabotegravir (30 mg) plus two NRTIs.

The study’s primary endpoint evaluated antiviral activity and safety through 32 weeks of maintenance therapy. At this time point, viral suppression rates for the two-drug regimen dosed every eight weeks (95%) or every four weeks (94%) were comparable with the rate observed in patients continuing with a three-drug oral regimen (91%).

Source: ViiV Healthcare, February 23, 2016

Pioglitazone for Stroke And Heart Attacks

Pioglitazone, a drug used to treat patients with type-2 diabetes, may prevent recurrent stroke and heart attacks in people with insulin resistance but without diabetes, according to results from the Insulin Resistance Intervention After Stroke (IRIS) trial, published in the New England Journal of Medicine. The study was supported by the National Institutes of Health’s National Institute of Neurological Disorders and Stroke.

The IRIS study was the first to provide evidence that a drug that targets cell metabolism may prevent secondary strokes and heart attacks before diabetes develops.

Stroke or heart attack occurred in 9% of participants receiving pioglitazone and in 12% of those given placebo, representing a relative decrease of 24%. The results suggest that 28 strokes or heart attacks may be prevented for every 1,000 patients who take pioglitazone for up to five years, according to the authors.

Sources: NIH, February 17, 2016, and NEJM, February 17, 2016

Aranesp for Myelodysplastic Syndrome

The phase 3 ARCADE study of darbepoetin alfa (Aranesp, Amgen) has met its primary endpoint of reducing the incidence of red blood cell transfusions in anemic patients with low- or intermediate-risk myelodysplastic syndrome (MDS) at the end of the 25-week study period. Darbepoetin also significantly improved the erythroid response, a key measure of the formation of new red blood cells.

Darbepoetin alfa is an erythropoiesis-stimulating protein that is produced in Chinese hamster ovary cells by recombinant DNA technology. It stimulates erythropoiesis by the same mechanism as endogenous erythropoietin.

Darbepoetin is indicated for the treatment of anemia due to chronic kidney disease, including patients on dialysis and patients not on dialysis. It is also indicated for the treatment of anemia in patients with nonmyeloid malignancies where anemia is due to the effect of concomitant myelosuppressive chemotherapy, and upon initiation there is a minimum of two additional months of planned chemotherapy.

Source: Amgen, February 16, 2016

Cell Therapy for Refractory RA

Mesoblast Ltd. has announced results from the first cohort of its ongoing phase 2 trial in rheumatoid arthritis (RA) patients who have previously failed one or more biologic agents. Preliminary results showed that a single intravenous infusion of the lower dose of the company’s mesenchymal precursor cell (MPC) product candidate MPC-300-IV (rexlemestrocel-L) was safe and resulted in early and sustained clinical responses. MPC-300-IV is being evaluated in a double-blind, randomized, placebo-controlled, dose-escalation trial in the U.S.

The study’s 12-week, prespecified efficacy endpoint (ACR20, a 20% improvement in RA using American College of Rheumatology response criteria) was achieved by 47% of all MPC-treated patients and by 60% of MPC-treated patients who had failed one or two biologics, compared with 25% and 17%, respectively, of matched placebo-treated controls. Seventy-one percent of MPC-treated patients who achieved ACR20 responses did so as early as week 1. At week 12, 27% of MPC-treated patients, but no placebo-treated controls, achieved ACR50 or ACR70 responses. Remission at week 12, as defined by a Disease Activity Score of less than 2.6, was seen in 20% of MPC-treated patients but in no controls.

Source: Mesoblast Ltd., February 16, 2016

Halaven for Liposarcoma

Positive results from a pivotal phase 3 study investigating the use of eribulin mesylate injection (Halaven, Eisai, Inc.) in certain types of soft-tissue sarcoma have been published online in the Lancet. The study evaluated overall survival (OS, the primary endpoint) in previously treated patients with advanced liposarcoma or leiomyosarcoma treated with eribulin compared with those treated with dacarbazine.

In the study, eribulin demonstrated a significant improvement in OS compared with dacarbazine. Median OS in all treated patients was 13.5 months with eribulin compared with 11.3 months with dacarbazine (hazard ratio, 0.75; P = 0.011). The treatment effects of eribulin were limited to patients with liposarcoma, according to preplanned subgroup analyses of OS.

Source: Eisai, February 10, 2016

Elagolix for Endometriosis

Positive data have been reported from the second of two replicate pivotal phase 3 trials evaluating the efficacy and safety of elagolix (AbbVie/Neurocrine Biosciences) in premenopausal women with pain from endometriosis. After six months of continuous treatment, both doses of elagolix (150 mg once daily and 200 mg twice daily) met the study’s coprimary endpoints. Elagolix reduced the scores for menstrual pain (dysmenorrhea) and nonmenstrual pelvic pain associated with endometriosis at three and six months, as measured by the Daily Assessment of Endometriosis Pain Scale.

Elagolix is an orally administered gonadotropin-releasing hormone antagonist that is being investigated in diseases that are mediated by sex hormones, such as uterine fibroids and endometriosis.

Source: AbbVie, February 10, 2016

Romosozumab for Osteoporosis

Top-line results have been announced from the phase 3, placebo-controlled Fracture Study in Postmenopausal Women With Osteoporosis (FRAME), which met its coprimary endpoints by reducing the incidence of new vertebral fractures through month 12 and month 24 in postmenopausal women with osteoporosis treated with romosozumab (Amgen UCB). The study also met its secondary endpoint of reducing the incidence of clinical fractures (a composite of vertebral and nonvertebral fractures) in postmenopausal women with osteoporosis through 12 months. However, the secondary endpoint of reducing the incidence of nonvertebral fractures through months 12 and 24 was not met.

Romosozumab is an investigational bone-forming monoclonal antibody. It is not approved by any regulatory authority for the treatment of osteoporosis. The drug inhibits the protein sclerostin and has a dual effect on bone, both increasing bone formation and decreasing bone breakdown.

Source: Amgen, February 22, 2016

Early Study Terminations

Rintega for Glioblastoma

Celldex Therapeutics discontinued a phase 3 study of rindopepimut (Rintega) in patients with newly diagnosed epidermal growth factor receptor vIII (EGFRvIII)-positive glioblastoma after a data safety and monitoring board determined that continuation of the study would not reach statistical significance for overall survival in patients with minimal residual disease, the study’s primary endpoint. Both the rindopepimut arm and the control arm were performing on par with each other. The median overall survival for rindopepimut was 20.4 months compared with 21.1 months for the control.

Rindopepimut is administered via intradermal injection and consists of the EGFRvIII-specific peptide sequence conjugated to keyhole limpet hemocyanin, a carrier protein. Rindopepimut stimulates the patient’s immune system, inducing pronounced EGFRvIII-specific humoral and cellular responses.

Source: Celldex Therapeutics, March 7, 2016

Ruxolitinib for Pancreatic Cancer

Incyte Corporation has announced its decision to discontinue a phase 2 study (JANUS-1) of ruxolitinib or placebo in combination with capecitabine for the second-line treatment of patients with advanced or metastatic pancreatic cancer. The decision to stop the study was made after a planned interim analysis demonstrated that ruxolitinib plus capecitabine did not show a sufficient level of efficacy to warrant continuation. Following these results, ongoing trials of ruxolitinib in solid tumors will be discontinued, including the phase 3 JANUS-2 trial in pancreatic cancer, a phase 2 substudy in patients with metastatic colorectal cancer and low C-reactive protein (CRP) levels, and phase 2 studies in breast and lung cancer.

Source: Incyte Corporation, February 11, 2016

DEVICE APPROVALS

Blazer Open-Irrigated Catheter

Boston Scientific has received FDA approval for the Blazer open-irrigated (OI) radiofrequency ablation catheter. The Blazer OI catheter has been approved for use in ablation procedures to restore a normal heart rhythm for patients in type I atrial flutter. During the procedure, localized electrical energy is delivered through an electrode on the tip of the catheter into the heart muscle, creating heat to destroy a small area of the tissue responsible for the abnormal heart rhythm.

Source: Boston Scientific, March 10, 2016

Test for Carbapenem Resistance Genes in “Superbugs”

Cepheid has received clearance from the FDA to market Xpert Carba-R, a qualitative in vitro diagnostic test for rapid, reproducible identification of five distinct families of carbapenem resistance genes that together represent the most common carbapenemases identified globally, including KPC, NDM, VIM, OXA-48, and IMP. The test is designed to be run on Cepheid’s GeneXpert system.

Xpert Carba-R is the first FDA-approved test for the detection and differentiation of carbapenemase genes in pure bacterial isolates previously shown to be non-susceptible to carbapenem antibiotics. These isolates may be cultured from a range of clinical specimens, including blood cultures, urine, respiratory samples, abscesses, and swab surveillance specimens.

Source: Cepheid, March 9, 2016

Automated Cervical Cancer Screening System

Becton, Dickinson and Company has received FDA approval for its BD Totalys MultiProcessor and BD Totalys SlidePrep instruments. Together with the BD Focal-Point SlideProfiler, these tools comprise the full BD Totalys system, which automates slide preparation, imaging, and review for use in cervical cancer screening, as well as providing ancillary-testing aliquot capability.

The BD Totalys system combined with the BD SurePath liquid-based Pap test offers an improved automated process for cervical cancer screening laboratories compared with current methods, according to the manufacturer.

Source: Becton, Dickinson and Company, March 7, 2016

Contact Lens Senses Changes in Eye Volume

The FDA has allowed the marketing of a one-time-use contact lens that may help practitioners identify the best time of day to measure a patient’s intraocular pressure (IOP). Elevated IOP is often associated with the optic nerve damage that is characteristic of glaucoma.

The Triggerfish consists of a sensor embedded in a soft silicone contact lens that detects tiny fluctuations in an eye’s volume. The device is worn for a maximum of 24 hours, transmitting data wirelessly from the sensor to an adhesive antenna worn around the eye. A portable data recorder worn by the patient receives information from the antenna and can transfer these data via Bluetooth to the clinician’s computer, which shows the range of time during the day that the pressure of the eye may be increasing. The device does not measure IOP; is not intended to be a diagnostic tool; and is not used to correct vision.

Source: FDA, March 4, 2016

Care Orchestrator Cloud-Based Software

Philips Respironics has announced that it received 510(k) clearance from the FDA for Care Orchestrator, its cloud-based clinical management software application.

Care Orchestrator is a first-of-its-kind connected health technology in the home care space that connects the portfolio of sleep and respiratory care, providing data, clinical management workflow, informatics, and intelligence for providers, payers, and patients within a single platform. Care Orchestrator is part of Philips’ HealthSuite, an open, cloud-based digital platform.

Source: Philips Respironics, March 1, 2016

Vacutainer UltraTouch Blood Collection Device

The FDA has given 510(k) clearance to a new blood collection device: the BD Vacutainer UltraTouch (Becton, Dickinson and Company).

The device employs the patented Penta Point Comfort five-bevel needle. When combined with BD RightGauge technology, which increases the needle’s inner diameter and allows clinicians to select a smaller-gauge needle without sacrificing sample quality and blood flow, the new device was shown to reduce penetration forces by up to 32% compared with another leading blood collection set, according to the manufacturer.

Source: Becton, Dickinson and Company, February 17, 2016

Scenergy CT–Ultrasound Fusion System

Clear Guide Medical has received 510(k) clearance from the FDA to market its computed tomography (CT)–ultra-sound fusion and image guidance system Scenergy. The device helps interventional radiologists and surgeons perform minimally invasive biopsies and other diagnostic and therapeutic procedures through an integrated display of fused ultrasound and CT images. The system is sold as an accessory to most ultrasound machines.

Source: Clear Guide Medical, February 16, 2016

FACSPresto Diagnostic Device for HIV/AIDS

Becton, Dickinson and Company has received FDA 510(k) clearance for its BD FACSPresto system and BD FACSPresto CD4/Hb cartridge.

The BD FACSPresto system is an automated multicolor fluorescent imaging cytometer and absorbance spectrometer that provides absolute and percentage results of CD4 T lymphocytes and hemoglobin (Hb) concentration in whole-blood samples to manage patients with human immunodeficiency virus (HIV) infection or acquired immune deficiency syndrome (AIDS).

Source: Becton, Dickinson and Company, February 10, 2016

Expression MR400 Monitor

Expression MR400 (Royal Phillips), a new technology that monitors patients undergoing magnetic resonance imaging (MRI), has received 510(k) clearance from the FDA. The MR400 device provides intensive care unit (ICU)-comparable monitoring in the MRI suite for all patients, including those who have serious medical conditions or who require anesthesia.

The device monitors the same vital signs that are tracked in the operating room and in the ICU, including heart rate, oxygen and carbon dioxide levels, body and surface temperatures, and blood pressure, and also includes advanced electrocardiogram monitoring.

Source: Royal Phillips, February 10, 2016

DynaMX Compression Staple

MX Orthopedics Corp. has announced FDA clearance of the dynaMX compression staple. The dynaMX line of fracture-fixation implants includes compression staples, screws, plates, and intramedullary implants. The dynaMX compression staples are made of superelastic nitinol to optimize biologic healing. The dynaMX line is engineered to generate and sustain higher levels of compression at the fracture site as the bone remodels, according to the manufacturer.

Source: MX Orthopedics Corp., February 10, 2016

OTHER DEVICE NEWS

Topas Device for Fecal Incontinence in Women

Endo is pulling out of the vaginal mesh business because of billions of dollars of litigation costs associated with the (often hazardous) devices for pelvic organ pro-lapse and urinary incontinence. But the company is still looking at its investigational mesh-based Topas device for the treatment of fecal incontinence in women.

The Topas device “does not require transvaginal incision, or any incision or modifications to the pelvic floor muscle, and thus has a different safety profile than the transvaginal mesh device,” the company said. The FDA’s Gastroenterology–Urology Devices Committee has unanimously voted that the device is safe and effective, making its approval likely.

Source: FierceMedicalDevices, March 8, 2016

Decipher Biopsy Test Launched for Prostate Cancer

GenomeDx Biosciences has announced the launch of Decipher Biopsy, a genomic test that evaluates RNA expression-based biomarkers to predict the risk of high-grade disease, metastasis, and cancer-specific death at the time of biopsy in men with prostate cancer. The launch brings the Decipher Prostate Cancer Classifier, already clinically adopted and validated following surgery, to newly diagnosed patients at the initial biopsy.

Decipher Biopsy is the only genomic test available to assess tumor aggressiveness across very low-, low-, intermediate-, and high-risk patients, enabling physicians to accurately determine if the patient may safely consider delaying initial local treatment or if he may benefit from earlier intensification of treatment with one or more therapies.

Source: GenomeDx Biosciences, March 8, 2016