Drugs
Emalex Biosciences says its lead candidate drug ecopipam, a novel dopamine-1 receptor antagonist, met its primary and secondary endpoints in a Phase 3 Tourette syndrome study. Company officials say they plan to meet with FDA to discuss submitting an NDA later this year for ecopipam to treat patients with
Drugs
Ascent Consumer Products is recalling one lot of its SinuCleanse Soft Tip Squeeze Bottle Nasal Wash System following a confirmed test result of microbial contamination involving Staphylococcus aureus (S. aureus). “Use of the SinuCleanse Soft Tip Squeeze Bottle Nasal Wash System contaminated with S. aureus can result in blood infections
Drugs
FDA has lifted a 12/2022-ordered clinical hold on Entrada Therapeutics’ Duchenne muscular dystrophy (DMD) therapy ENTR-601-44 and provided authorization to begin ELEVATE-44-102, a Phase 1b multiple ascending dose clinical study. The study of ENTR-601-44 in adult patients involves those with a confirmed
Drugs
Former CDER director Patrizia Cavazzoni is returning to her industry roots and rejoining Pfizer as the company’s chief medical officer. Cavazzoni spent seven years in CDER’s management, including the past four years as Center director after replacing Janet Woodcock who moved to a more senior role in the
Drugs
FDA has approved a Medtronic PMA for its BrainSense Adaptive deep brain stimulation (aDBS) and BrainSense Electrode Identifier (EI) for use in Parkinson’s patients. The device transmits electrical signals to specific parts of the brain affected by debilitating neurological disorders, like Parkinson’s. The BrainSense Adaptive technology is designed
Drugs
In a move that is attempting to reverse some of the damage to public health safety and regulatory reviews of innovative products, FDA is rehiring up to 300 scientists and reviewers, which is less than a third of about 1,000 agency staff that were terminated last week as part
Drugs
FDA has told Insmed that it does not currently plan to hold an advisory committee meeting to discuss the company’s NDA for brensocatib for patients with non-cystic fibrosis bronchiectasis. Insmed says brensocatib is the first in a new class of medicines called dipeptidyl peptidase 1 (DPP1) inhibitors for
Drugs
FDA has accepted for priority review a Bristol Myers Squibb supplemental BLA for Opdivo (nivolumab) plus Yervoy (ipilimumab) for a first-line treatment option for adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (mCRC). The agency set a
Drugs
Drug compounding operations have filed suit against FDA in northern Texas federal court, seeking an order to reverse the agency decision last week to remove semaglutide (Novo Nordisk’s Wegovy and Ozempic) from the drug shortage list (see earlier story). “Arbitrarily dismissing evidence that the shortage persists, FDA removed semaglutide
Drugs
FDA has determined the shortage of semaglutide injection products (Novo Nordisk’s Wegovy and Ozempic) is resolved. “FDA confirmed with the drug’s manufacturer that their stated product availability and manufacturing capacity can meet the present and projected national demand,” an agency notice says. “Patients and prescribers may still see
Drugs
Pfizer has decided to discontinue the marketing of its hemophilia B gene therapy Beqvez (fidanacogene elaparvovec-dzkt) due to limited patient and physician interest in the $3.5 million one-time treatment. The product is intended for treating adults with moderate to severe hemophilia B who currently use factor IX
Drugs
FDA has accepted for priority review a Boehringer Ingelheim NDA for zongertinib (BI 1810631) for treating adult patients with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have HER2 (ERBB2) mutations and who have received prior systemic therapy. The user fee review action target date is expected
Drugs
FDA has approved a Mirum Pharmaceuticals NDA for Ctexli (chenodiol) for treating adults with cerebrotendinous xanthomatosis (CTX), a very rare lipid storage disease. The genetic metabolic disorder is caused by a mutation in a gene called CYP27A1, “resulting in a deficiency of the enzyme that is important in the body’
Drugs
Pharmaceutical Research and Manufacturers of America (PhRMA) says FDA should revise its recommendations for sponsors requesting meetings concerning cellular and gene therapy products (CGTs) to better align with the drug user fee program’s commitment letter and existing agency procedures. PhRMA suggests this in just-posted comments on a draft
Drugs
CBER deputy director Celia M. Witten has left the agency, the latest high-ranking FDA official to depart as staff morale plunges in the wake of Trump Administration firings of probationary employees that began earlier this week (see story). According to a just-posted organization chart, Witten was one of
Drugs
Three Arnall Golden Gregory attorneys say that foreign drug and medical device companies are likely to see heightened FDA enforcement activity as part of the second Trump administration’s “America First” agenda. Writing in an online post, the attorneys present statistics on FDA overseas inspections before and after the Covid-
Drugs
Sens. Chuck Grassley (R-IA) and Amy Klobuchar (D-MN) have reintroduced their Safe and Affordable Drugs From Canada Act to “allow Americans to safely import prescription drugs from Canada — lowering costs, increasing access, and strengthening competition in the pharmaceutical market.” A statement issued by Grassley includes a link to
Drugs
Partnership for Safe Medicines (PSM) has released a new report that found “suspicious, unauthorized, and illegal ingredients for popular diabetes and obesity injectables (commonly known as weight loss drugs) are flooding into the U.S. from foreign sources despite U.S. laws forbidding them from coming through the border.” Despite
Drugs
Supernus Pharmaceuticals is reporting that a Phase 2b study of SPN-820 in adults with treatment-resistant depression did not demonstrate a statistically significant improvement on the primary endpoint of change from baseline in the Montgomery-Åsberg Depression Rating Scale total score to Week 4. “There was no treatment difference
Drugs
FDA has approved an expanded access program authorizing ImmunityBio to provide recombinant BCG to urologists to address the TICE BCG shortage to treat bladder cancer. A company statement says the alternative BCG source was developed by the Serum Institute of India. Recombinant BCG (Bacillus Calmette-Guérin) is described as a
Biologics
In a Hyman, Phelps & McNamara FDA Law Blog post, principal regulatory device and biologics expert Richard Lewis, who was an FDA reviewer and compliance officer during the 2018-2019 five-week government shutdown, describes what such a funding gap means for the agency and its regulated industries. The post
Drugs
FDA has issued a refusal to file letter for Harmony Biosciences’ Wakix (pitolisant) to treat excessive daytime sleepiness in adults with idiopathic hypersomnia. A company statement says the company plans to continue with a pivotal Phase 3 study based on FDA input. “We believe this study design will effectively demonstrate