This week saw a rejection by the FDA for Aradigm, a clinical hold for Bellicum, and a black box warning for Intercept. In addition, the FDA granted a Breakthrough Therapy designation along with a Fast Track designation. Here are the top regulatory stories for the week:
The FDA has rejected Aradigm’s inhaled antibiotic Linhaliq and wants a new Phase III trial that can provide evidence of two years or more of durability in preventing “the co-primary endpoints of frequency and severity of exacerbations.” https://endpts.com/aradigm-completes-disastrous-regulatory-run-as-fda-slaps-down-linhaliq-application/?utm
Bellicum reported that the FDA had slapped a clinical hold on its lead cell therapy after researchers tracked three cases of encephalopathy among patients taking the drug to support allogeneic stem cell transplants. https://endpts.com/brain-damage-cases-force-an-fda-hold-on-bellicums-lead-cell-therapy/?utm
CutisPharma announced that the FDA has approved Firvanq (vancomycin hydrochloride) for oral solution, for the treatment of Clostridium difficile (C diff) associated diarrhea and enterocolitis. https://www.drugs.com/newdrugs/cutispharma-announces-fda-approval-firvanq-vancomycin-clostridium-difficile-associated-diarrhea-4687.html?utm
Alkermes announced that it has submitted a NDA to the FDA for ALKS 5461, a once-daily, oral investigational medicine with a novel mechanism of action for the adjunctive treatment of major depressive disorder. https://www.drugs.com/nda/alks_5461_180131.html?utm_source=ddc&utm
Alnylam Pharmaceuticals announced that the FDA has accepted its NDA for Patisiran, an investigational RNAi therapeutic targeting transthyretin for the treatment of hereditary ATTR (hATTR) amyloidosis. The FDA also granted the Company’s request for Priority Review and has set a PDUFA date of August 11, 2018. http://investors.alnylam.com/news-releases/news-release-details/alnylam-announces-fda-acceptance-new-drug-application-nda-and
Roche said the FDA awarded Breakthrough Therapy Designation to its experimental autism treatment balovaptan (RG7314). A vasopressin 1a (V1a) receptor antagonist, balovaptan has shown the potential to improve social interaction and communication in people with autism spectrum disorder. https://www.biospace.com/article/unique-fda-calls-roche-s-autism-drug-a-breakthrough?utm
Corbus Pharmaceuticals announced an agreement with FDA on its Phase 2b study of lenabasum (formerly known as anabasum) in the treatment of cystic fibrosis with pulmonary exacerbations as the sole primary endpoint https://www.drugs.com/clinical_trials/corbus-pharmaceuticals-announces-agreement-fda-phase-2b-cystic-fibrosis-study-design-pulmonary-17708.html?utm
Sunovion said that it plans to file for FDA approval in the spring for its experimental Parkinson’s drug, APL-130277 which succeeded in a 109-patient Phase 3 trial. The drug helped improve the motor function of adult Parkinson’s patients suffering “off” episodes, meaning their medications aren’t working. https://www.xconomy.com/boston/2018/01/30/sunovion-parkinsons-drug-heads-to-fda-setting-up-battle-with-acorda/
Takeda’s Zika vaccine candidate TAK-426 received FDA fast track designation https://www.fiercepharma.com/vaccines/takeda-s-zika-vaccine-gets-fda-fast-track-though-virus-no-longer-emergency
Aquestive Therapeutics (formerly Monosol) announced that the FDA has granted orphan drug designation to Riluzole Oral Soluble Film (riluzole OSF) for the treatment of ALS https://www.firstwordpharma.com/node/1540734
The FDA said it would add a black box warning to the label of Intercepts’ chronic liver disease drug Ocaliva, cautioning against overdosing higher-risk patients and recommending closer monitoring. https://www.biopharmadive.com/news/fda-adds-black-box-warning-to-intercepts-ocaliva/516115/
Three companies (AMAG, Gilead, and Vertex) are expecting FDA decisions in February for key programs: https://www.biospace.com/article/unique-3-fda-decisions-to-look-out-for-in-february/?utm
The EMA released updated procedural advice on how it evaluates advanced therapy medicinal products (ATMPs) such as cell and gene therapies. https://www.raps.org/news-and-articles/news-articles/2018/2/ema-updates-on-advanced-therapy-evaluations,-risk
In other news…
Just days after Vical announced that their late-stage study of the CMV vaccine ASP0113 had failed badly, the San Diego-based biotech is cutting more than half of its staff: https://endpts.com/counting-its-remaining-cash-vical-axes-40-staffers-and-circles-wagons-around-two-phase-ii-assets/?utm
Sanofi entered into a definitive agreement to acquire Ablynx for 45 euros ($55.79) per share in cash, or approximately $4.8 billion https://www.firstwordpharma.com/node/1539976
Canada-based Novelion Therapeutics will slash an unknown number of jobs as it initiates “significant cost reduction plans.” https://www.biospace.com/article/unique-novelion-to-cut-an-unknown-number-of-jobs-in-restructuring/?utm
Pfizer released an update of its pipeline, confirming cuts to eight neuroscience projects and also cutting four oncology programs: https://www.biospace.com/article/unique-pfizer-confirms-neuroscience-cuts-shelves-another-4-early-stage-projects?utm
Seattle Genetics will pay $614 million, for Cascadian Therapeutics and its lead drug tucatinib, being developed for women with HER2-positive metastatic breast cancer. https://www.xconomy.com/seattle/2018/01/31/bio-buyout-spree-continues-as-seagen-bets-614m-on-cascadian-cancer-drug/
Takeda and its partner Zinfandel Pharmaceuticals announced that their global Phase III trial has been terminated after showing inadequate treatment effect in delaying the onset of mild cognitive impairment due to Alzheimer’s disease. https://www.drugs.com/clinical_trials/takeda-zinfandel-pharmaceuticals-discontinue-tommorrow-trial-following-planned-futility-analysis-17710.html?utm
Four months after the FDA declined to approve its lead diabetes treatment due to manufacturing concerns, Intarcia Therapeutics has laid off 60 employees and terminated two late-stage trials of the drug-device https://www.bizjournals.com/boston/news/2018/02/02/biotech-unicorn-intarcia-hit-by-layoffs-trial.html
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