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Vertex doesn’t need to do any more safety studies to identify potential off-target effects for exa-cel (exagamglogene autotemcel), its investigational gene-editing therapy designed to cure sickle cell disease, an FDA advisory committee decided. Read More
Despite missing its pivotal phase 3 primary efficacy endpoint, Sarepta is sailing confidently into a supplemental application to remove the age limitation on Elevidys (delandistrogene moxeparvovec-rokl), its gene therapy for children with Duchenne muscular dystrophy (DMD). Read More
Whether Vertex Pharmaceuticals’ off-target safety analysis of its investigational sickle cell gene therapy exagamglogene autotemcel (exa-cel) is sufficient or not is the issue before an FDA advisory committee on Tuesday as it considers the therapy. Read More
The FDA’s guidance on developing topical corticosteroids provides ANDA applicants with recommendations on pilot and pivotal studies to assess bioequivalence of the drugs. Read More
In this edition of Quick Notes, we cover a Philips study confirming the safety of its cardiac diagnostic device, FTC action against deceptive marketing of a COVID-19 protection device, and updates to two FDA medical device event reporting databases. Read More
The FDA issued a complete response letter (CRL) for Sanofi and Regeneron’s supplemental biologics license application (sBLA) for Dupixent (dupilumab) in chronic spontaneous urticaria (CSU) requesting additional efficacy data, the companies announced on Friday. Read More
In this edition of Quick Notes, we cover a pharmaceutical ingredient cartel, fake Ozempic pens and new transparency rules for the European Medicines Agency clinical trial information system. Read More
After an FDA advisory committee voted overwhelmingly against its ALS drug NurOwn, BrainStorm Cell Therapeutics announced that it is withdrawing the BLA for the drug so that it can conduct an additional phase 3 trial. Read More
It’s rare, but it turns out a company seeking approval for a new indication can find success in the wake of an FDA Complete Response Letter (CRL) as is the case for Ardelyx’s Xphozah (tenapanor). Read More
The FDA has issued a new final guidance intended to facilitate the development and assessment of regenerative medicine therapy (RMT) products CBER regulates through identifying and recognizing voluntary consensus standards (VCS). Read More
Seeking to better understand innovative clinical trial designs and approaches, CDER has asked stakeholders to share feedback on current barriers and opportunities around this topic. Read More