Archive for October 2022

The FDA clarifies its regulatory requirements for small entity establishments that manufacture human cells, tissues, or cellular or tissue-based products (HCT/P), in a final guidance released today. Source: Drug Industry Daily

Due to a provision in the recently passed Inflation Reduction Act (IRA), Alnylam Pharmaceuticals won’t be conducting a phase 3 trial of its RNAi therapeutic to treat the rare eye condition Stargardt disease. Source: Drug Industry Daily

In a draft guidance released today, the FDA says trials of drugs for pediatric use should train clinical investigators and trial staff to measure and evaluate the drug’s potential impact on participants’ physical growth and sexual development. Source: Drug Industry Daily

The FDA has issued a Deferred Action letter for Amicus Therapeutics’ biologic license application (BLA) for cipaglucosidase alfa, the biologic component of AT-GAA, its investigational dual therapy for muscle-wasting Pompe disease. Source: Drug Industry Daily

The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 16-0 on Friday against recommending Y-mAbs Therapeutics’ biologic candidate for the treatment of central nervous system/leptomeningeal (CNS/LM) metastases in children with neuroblastoma who have had the standard treatment for CNS disease. Source: Drug Industry Daily

The FDA has rejected a citizen petition pressing the agency to revoke the accelerated approval for Jazz Pharmaceuticals’ and PharmaMar’s Zepzelca (lurbinectedin), a treatment for adults with metastatic small cell lung cancer (SCLC). Source: Drug Industry Daily

Older people, smokers and those with an increased risk of cardiovascular problems shouldn’t use Janus kinase inhibitors for chronic inflammatory diseases unless there are no other suitable treatment options, according to new recommendations by the European Medicines Agency (EMA). Source: Drug Industry Daily

The FDA has declined to green-light Gilead Sciences’ hepatitis delta virus (HDV) drug bulevirtide because of manufacturing and delivery concerns, meaning there will continue to be no approved treatment for the condition in the U.S. Source: Drug Industry Daily

As the FDA’s decision date on GSK’s new drug application (NDA) for daprodustat — its chronic kidney disease anemia drug candidate — approaches, an FDA advisory panel said Wednesday that it favors the drug for kidney patients on dialysis but not for patients who aren’t. Source: Drug Industry Daily

The FDA is falling under the political spell that “more drugs, and faster” is the best way to serve the healthcare needs of American citizens, according to a new report that also criticizes the agency’s lack of transparency and reliance on industry funding. Source: Drug Industry Daily