Drugs
Harmony Biosciences says topline results from its Phase 3 registrational clinical trial (RECONNECT study) of ZYN002 in Fragile X syndrome (FXS) did not meet the primary endpoint of improvement in social avoidance primarily due to a higher-than-expected placebo response rate. “Although the study did not achieve its primary
Drugs
Acadia Pharmaceuticals says it will cease developing intranasal carbetocin after its Phase 3 COMPASS PWS trial of the drug for treating hyperphagia in patients with Prader-Willi syndrome (PWS) did not achieve its primary or secondary endpoints. The 12-week, randomized, placebo-controlled study enrolled 175 children and adults ages
Drugs
Reps. August Pfluger (R-TX) and Greg Landsman (D-OH) have introduced the Biosimilar Red Tape Elimination Act, which they say would improve patient access to lower-cost prescription medication and increase competition in the biological drug market by automatically deeming biosimilar drugs as interchangeable with their branded counterparts upon
Drugs
uniQure says its investigational gene therapy AMT-130 significantly slowed disease progression in Huntington’s disease patients, which the company calls the most convincing evidence to date of a disease-modifying effect in the fatal neurodegenerative disorder. In a pivotal Phase 1-2 study, patients who received a high dose
Drugs
An FDA review of records submitted by Chengdu Brilliant Biopharmaceutical Co. in Shuangliu District, Chengdu, Sichuan, China, in response to an agency request, found significant deviations from current good manufacturing practice (CGMP) regulations for active pharmaceutical ingredients (APIs). A 9/11 Warning Letter says the specific deviations were: * failure to
Drugs
FDA has issued Scholar Rock a complete response letter for its BLA seeking approval of apitegromab, a first-in-class muscle-targeted therapy for spinal muscular atrophy (SMA). The letter cited observations made during an FDA inspection of Catalent Indiana, a third-party fill-finish facility acquired by Novo Nordisk
Drugs
FDA has released the form FDA-483 issued following a 1/9-1/17 inspection at the Genzyme Ireland drug manufacturing facility in Waterford, Ireland. The two inspection observations were: * failure to establish procedures, processes, and testing designed to prevent microbial contamination of the drug product purporting to be sterile;
Drugs
FDA has released the form FDA-483 issued following an 8/4-8/12 inspection at the Cohance Lifesciences drug manufacturing facility in Secunderabad, Telangana, India. The inspection observations were: * failure to thoroughly review any unexplained discrepancy and the failure of a batch or any of its components to meet
Drugs
Sens. Rick Scott (R-FL) and Kirsten Gillibrand (D-NY), the chairman and ranking member of the Senate Aging Committee, have asked FDA commissioner Martin Makary for information about the agency’s oversight of foreign drug manufacturing and how it may threaten the quality and safety of drugs marketed and
Drugs
FDA has issued Biogen a complete response letter on a supplemental NDA for a high-dose regimen of Spinraza (nusinersen) for treating spinal muscular atrophy (SMA). The letter requested an update to the technical information included in the Chemistry Manufacturing and Controls module of the submission, according to the company.
Drugs
FDA has extended by three months its review of Sanofi’s multiple sclerosis drug tolebrutinib in response to additional analyses submitted during the ongoing NDA review. The agency determined that the new data constituted a “major amendment,” triggering the extension. The revised user fee review action target date is 12/
Drugs
Roche says its investigational breast cancer drug giredestrant met key goals in a pivotal Phase 3 study, showing significant benefits for patients with advanced estrogen receptor (ER)-positive disease. The evERA trial evaluated giredestrant in combination with everolimus against standard-of-care endocrine therapy plus everolimus in patients with ER-
Drugs
FDA has released a draft guidance entitled Malaria: Developing Drugs for Treatment that outlines key considerations for companies developing drugs to treat the tropical disease. The document is intended to help drugmakers design clinical trials for therapies targeting malaria caused by a range of Plasmodium species, including P. falciparum, the
Drugs
Lexicon Pharmaceuticals says FDA is requiring additional time to review its new clinical data submitted earlier this month in an effort to advance its stalled NDA for its diabetes drug Zynquist (sotagliflozin). The company is seeking to address concerns raised in a 12/2024 complete response letter that cited an
Drugs
FDA has accepted for priority review a Chiesi NDA for idebenone and its use in treating Leber hereditary optic neuropathy (LHON), a rare inherited condition that causes sudden and severe central vision loss. LHON is a mitochondrial disorder that damages retinal ganglion cells, leading to rapid vision decline, typically beginning
Drugs
FDA has granted accelerated approval to a Stealth Biotherapeutics NDA for Forzinity (elamipretide) injection for treating Barth syndrome, a rare and often fatal mitochondrial disease that primarily affects boys. The disease typically begins with severe heart failure in infancy, significantly reducing life expectancy and impairing quality of life, an agency
Drugs
FDA has rejected Otsuka Pharmaceutical and H. Lundbeck’s bid to expand the use of Rexulti (brexpiprazole) for treating post-traumatic stress disorder in adults. An agency-issued complete response letter said the application, which sought approval for Rexulti in combination with the antidepressant sertraline (Zoloft), did not provide “substantial
Drugs
FDA late 9/22 took two actions against autism, first ordering a label change for acetaminophen (Tylenol) to reflect evidence suggesting that its use by pregnant women may be associated with an increased risk of neurological conditions such as autism and attention deficit in children, and second approving leucovorin calcium
Drugs
FDA has approved Merck’s pembrolizumab and berahyaluronidase alfa-pmph (Keytruda Qlex) for subcutaneous injection for adult and pediatric (12 years and older) solid tumor indications approved for the intravenous formulation of pembrolizumab. Approval was based on data from the Study MK-3475A-D77 (NCT05722015), a randomized, multicenter, open-label,
Drugs
Regeneron says it will apply to FDA by the end of 2025 for approval of its garetosmab to treat the ultra-rare genetic disorder fibrodysplasia ossificans progressiva (FOP). A company statement says the application will be based on the results of the Phase 3 OPTIMA trial that demonstrated that garetosmab
Drugs
In a white paper explaining FDA’s 9/12 guidance on using alternative tools to assess drug manufacturing facilities identified in pending applications, three Sidley attorneys say U.S. companies “can and should leverage the flexibility of these alternative tools to streamline regulatory timelines and approval pathways.” They also say
Drugs
FDA has released the form FDA-483 issued following a 7/29-8/8 inspection at Fagron Compounding Services, a Wichita, KS, outsourcing facility. The six inspection observations were: * failure to establish and follow written procedures to prevent microbiological contamination of drug products purporting to be sterile; * failure to thoroughly