Drugs
Is biotech G1 Therapeutics, which was cofounded by FDA acting commissioner Ned Sharpless, getting preferential treatment for its cancer drug trilaciclib? Progressive watchdog group Restore Public Trust says something smells fishy after the drug was recently given “breakthrough status” and company officials have reportedly been told by FDA to prepare
Drugs
FDA says it is planning an 11/7 public meeting on “Promoting Effective Drug Development Programs: Opportunities and Priorities for FDA’s Office of New Drugs” to obtain “actionable policy suggestions“ for CDER review staff “regarding opportunities to promote effective drug development while maintaining the FDA’s regulatory standards for
Drugs
FDA has sent Kala Pharmaceuticals a complete response letter (CRL) on its NDA for KPI-121 (loteprednol etabonate) 0.25% for the temporary relief of dry eye disease signs and symptoms. The agency letter recommended efficacy data from an additional clinical trial before resubmission, the company says. Kala says it
Drugs
A new FDA Impact Story discusses new research the agency has undertaken to help would-be generic topical dermatological drug product developers demonstrate bioequivalence. “FDA collaborated with international researchers to evaluate a new method for monitoring the amount of a topical drug in the dermis,” it says. “In a procedure
Drugs
FDA has accepted for review an Ortho Dermatologics NDA for tazarotene 0.045%) lotion for treating moderate to severe acne. The agency has set a 12/22 user fee review action target date. The submission includes data from two Phase 3 trials in 1,614 patients. The primary efficacy endpoints
Drugs
Amarin says it expects FDA will extend its review of a supplemental NDA seeking an expanded indication for Vascepa (icosapent ethyl) capsules based on data from the REDUCE-IT cardiovascular (CV) outcomes study. The current user fee review action target date is 9/28, but FDA told the company 8/
Drugs
Following a 7/25 meeting with FDA, Evoke Pharma says it will resubmit its Gimota (metoclopramide) NDA to the agency in the fourth quarter of 2019. The company says the meeting was to obtain FDA feedback and agreement on the company’s plan to address deficiencies cited in a 4/
Drugs
FDA has granted Dynacure an orphan drug designation for DYN101, an investigational antisense medicine designed to modulate the expression of dynamin 2 (DNM2) for treating centronuclear myopathies (CNM). The company expects to initiate a Phase 1/2 study (Unite-CNM) in a few months. The therapy is being developed in
Drugs
The Federal Trade Commission (FTC) says Boston Scientific has agreed to divest certain assets to Varian Medical Systems to resolve FTC charges that the company’s proposed $4.2 billion acquisition of BTG would violate antitrust law. BTG is a medical equipment and pharmaceutical supplier. FTC says that as originally
Drugs
CDER director Janet Woodcock says that rather than develop individual products, the National Institutes of Health (NIH) could be of greatest use by filling the gaps in translational science generally to help understand how to evaluate health conditions. Speaking at a 7/25 workshop on the NIH role in drug
Drugs
FDA has authorized a Helix BioPharma IND to begin a Phase 1b/2 study of L-DOS47 and doxorubicin in patients with advanced metastatic pancreatic cancer who have progressed on at least two prior treatment regimens. The Phase 1b will involve a dose escalation safety run-in to determine the
Drugs
Sarepta Therapeutics says an adverse report that was erroneously submitted to the FDA adverse event reporting system on a case of rhabdomyolysis in a participant in a study of the company’s micro—dystrophin gene therapy candidate in patients with Duchenne muscular dystrophy did not result in any interruption in
Drugs
FDA has released the FDA-483 issued following a 7/24-8/2 inspection at AveXis, San Diego, CA, the control testing laboratory at the center of data manipulation concerns (see story) with recently approved Zlogensma, a gene therapy product intended to treat children less than two years old with
Drugs
FDA’s Antimicrobial Drugs Advisory Committee has voted 16-2 to recommend approval for Gilead Sciences’ HIV drug Descovy (emtricitabine 200 mg and tenofovir alafenamide 25 mg tablets; F/TAF) for pre-exposure prophylaxis (PrEP) in men and transgender women who have sex with men. Panel members were mixed in
Drugs
Senate Finance Committee chairman Chuck Grassley (R-IA) says FDA should reinstate unannounced inspections of prescription drug manufacturing facilities in foreign countries in light of the administration’s proposed Safe Importation Action Plan and the fact that the foreign facilities provide most of the ingredients for drug production in the
Drugs
FDA has issued a draft guidance, Fabry Disease: Developing Drugs for Treatment, to give sponsors recommendations regarding clinical trial design features that can support approval of drugs and biological products intended to treat Fabry Disease (FD). The document says FD is a rare, X-linked, slowly progressive, lysosomal storage disorder
Drugs
The CDER Office of Prescription Drug Promotion is proposing research to determine healthcare providers’ awareness, perceptions, and beliefs about the benefits and risks of an FDA-approved product that carries a Boxed Warning. A Federal Register notice says Boxed Warning information may be included as part of prescribing information at
Drugs
FDA is funding a 9/17 meeting hosted by The Critical Path Institute and the National Organization for Rare Disorders (NORD) to formally launch development of a new rare disease data and analytics platform. The host organizations say the platform’s goal is to accelerate movement of therapies from bench
Drugs
The Government Accountability Office (GAO) says FDA should take additional steps to address factors that may affect approval rates of generic drugs in the first review cycle. A GAO report says that 12% of the 2,030 generic drug applications reviewed by FDA from FY 2015 through FY 2017 were
Drugs
FDA has accepted for priority review a Blueprint Medicines NDA for avapritinib for treating adult patients with PDGFRA Exon 18 mutant gastrointestinal stromal tumors (GIST), regardless of prior therapy, and fourth-line GIST. The agency set a user fee review action target of 2/14/2020. Avapritinib is an investigational,
Drugs
FDA has accepted for review an Alnylam Pharmaceuticals NDA for givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for treating acute hepatic porphyria. The agency has granted a priority review and set a 2/4/2020 user fee review action target date. Givosiran is intended to lower
Drugs
The FDA annual report on drug and biologic postmarketing requirements (PMRs) and postmarketing commitments (PMCs) for FY 2018 shows that as of 9/30/18 there were 318 unique applicants with open PMRs/PMCs under 755 unique NDAs and BLAs. In the previous fiscal year, there were 299 unique applicants