The FDA has revised the Manual of Policies and Procedures (MAPP) for ANDA assessment practices with changes intended to decrease the number of review cycles needed for approval. Read More

Alnylam Pharmaceuticals today announced that the FDA has issued a complete response letter (CRL) on its supplemental New Drug Application (sNDA) for Onpattro (patisiran) for the treatment of cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis and will no longer pursue the expanded indication. Read More

Amgen’s open-label confirmatory study of its lung cancer drug Lumakras (sotorasib) had too many data integrity problems to effectively show that the drug is an improvement over the standard-of-care chemotherapy docetaxel, according to the Oncologic Drugs Advisory Committee (ODAC), which met Thursday. Read More

US WorldMeds got a 14-6 thumbs-up from FDA’s Oncologic Drugs Advisory Committee, supporting DFMO (difluoromethylornithine) to reduce the risk of relapse in children in remission after multifaceted treatment for high-risk neuroblastoma. Read More

Citing deficiencies in the Chemistry, Manufacturing, and Controls (CMC) processes, FDA handed Swiss drugmaker Galderma a complete response letter for RelabotulinumtoxinA, the company’s cosmetic neurotoxin intended to treat frown lines and crow’s feet. Read More

Despite strong testimony and comments from patients and clinicians, the FDA advisory committee reviewing NurOwn, an investigational treatment for amyotrophic lateral sclerosis (ALS), voted against recommending the drug, citing a lack of consistent data and ill-defined biomarkers. Read More

This European Edition of Quick Notes includes guidelines from WHO for pharmaceutical quality control laboratories, UK launch of the Innovative Devices Access Pathway pilot, European Commission (EC) unannounced antitrust inspections at a medical device company and an open letter to the EC about MDR and IVDR. Read More