Drugs
FDA has approved Chiesi Global Rare Diseases’ Lamzede (velmanase alfa-tycv) to treat non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients. The company says Lamzede is a recombinant form of human alpha-mannosidase intended to provide or supplement natural alpha-mannosidase, an enzyme that is
Drugs
FDA has published a draft guidance, Product-Specific Guidance Meetings Between FDA and ANDA Applicants Under GDUFA, with information on requesting and conducting product-specific guidance (PSG) meetings with FDA as contemplated in the GDUFA 3 reauthorization commitment letter. The document says the guidance is intended to provide procedures that
FDA Policy/General
FDA commissioner Robert Califf says the agency will be tackling the problem of misleading health information, which he views as a “leading cause of preventable death in America now.” Califf made the comment in a paper marking the first anniversary of his becoming the agency’s 25th commissioner. He said
Drugs
FDA has approved Apellis Pharmaceuticals’ Syfovre (pegcetacoplan injection) to treat geographic atrophy (GA) secondary to age-related macular degeneration. The company says Syfovre is the only FDA-approved treatment for GA. The approval was based on positive results from the Phase 3 OAKS and DERBY studies at 24 months across
Drugs
Health policy researchers in the UK say their analysis of drug company financial statements shows that company claims that high drug prices are needed to provide money for research and development into new drugs don’t hold up. Writing in thebmj, they call for government intervention or regulation to incentivize
Drugs
FDA commissioner Robert Califf says that as the agency considers ways to implement recommendations in a report evaluating its efforts to address the nation’s opioid crisis, it is “already moving forward on multiple workstreams, while also adapting to the changing epidemiology of the ongoing public health emergency.” Califf’s
Drugs
FDA has published 30 new product-specific guidances (PSGs) with recommendations for developing generic drugs and generating evidence to support ANDA approvals. The agency PSG Web page has links to the lists of 21 new and nine revised PSGs. There are 21 PSGs for products with no approved ANDAs, including
Drugs
Pfizer says data from the Phase 3 TALAPRO-2 study show a statistically significant and clinically meaningful 37% reduction in risk of disease progression or death in men with metastatic castration-resistant prostate cancer treated with Talzenna (talazoparib) plus Xtandi (enzalutamide). The company also reported that FDA has granted priority
Drugs
FDA has approved a BLA supplement submitted 8/12/22 by Amgen’s BioVex unit to update the labeling and Medication Guide for talimogene laherparepvec, a melanoma treatment. The update reflects herpetic infection, including disseminated herpetic infection, as an adverse drug reaction with talimogene laherparepvec administration, FDA says.
Drugs
While FDA historically stays out of debates about drug prices and it has no legal authority to investigate or control such prices, it is now being tasked to work with CMS’ Innovation Center to test a proposed model that would adjust Medicare payments for accelerated approval program (AAP) drugs. “Working
Biologics
The FDA MCMi Program Update annual report describes the agency’s efforts to ensure that medical countermeasures (MCMs), such as drugs, vaccines, and diagnostic tests, are safe, effective, and secure as they protect the U.S. from chemical, biological, radiological, nuclear (CBRN), and emerging infectious disease threats. The report says
Drugs
After a review of Concert Pharmaceuticals’ deuruxolitinib clinical data, FDA has determined that the drug will maintain its breakthrough therapy designation to treat adults with moderate to severe alopecia areata. Deuruxolitinib is described as an oral Janus kinase (JAK) inhibitor. A Concert statement says that in light of the recent
Drugs
FDA has accepted for priority review a Galera Therapeutics NDA for avasopasem manganese for radiotherapy-induced severe oral mucositis (SOM) in patients with head and neck cancer undergoing standard-of-care treatment. The agency has set an 8/9 user fee review action target date. The submission is based on
Drugs
A Foley Hoag online product liability update explains a Massachusetts federal court’s actions in a case in which a plaintiff sued the manufacturer and distributor of a drug used to treat Cushing’s disease, alleging that it caused ovarian cysts and uterine bleeding that required surgical dilation and curettage
Drugs
Researchers from several organizations and institutions say the use of extrapolation from adult to pediatric drugs between 1/1/2015 and 7/31/2021 increased the rates of new and expanded indications in the period. Writing in the Journal of Biopharmaceutical Statistics (purchase required), the researchers say that within the
Drugs
With FDA remaining silent on orphan drug exclusivities for products approved after the 2021 11th Circuit Court of Appeals decision in Catalyst Pharmaceuticals v. Becerra, there is a backlog of exclusivity decisions awaiting FDA action. That’s the report of attorneys Jacqueline Berman and Nikita Bhojani (Morgan Lewis) writing in
Drugs
A joint FDA Nonprescription Drugs Advisory Committee and the Anesthetic and Analgesic Drug Products Advisory Committee Meeting unanimously voted 2/15 to recommend the nonprescription use of Emergent BioSolutions’ Narcan (naloxone HCl) nasal spray, 4 mg/.1 mL, for treating known or suspected opioid overdose. The 19-0 vote came
Drugs
FDA has issued Soligenix a refuse-to-file letter for its HyBryte (synthetic hypericin) NDA that is seeking an indication for treating early stage cutaneous T-cell lymphoma. The letter says the NDA, which was submitted 12/14/2022, was not sufficiently complete to allow the review. The company says
Drugs
A 9/12-9/16/22 FDA inspection at the Delta Kozmetik Sanayi Ve Ticaret-Selim Yesil drug manufacturing facility in Tuzia/Istanbul, Turkey, identified significant violations of current good manufacturing practice (CGMP) regulations for finished drugs. A 2/13 Warning Letter says the specific violations were: * failing to test
Drugs
Six Republican senators and five GOP House members have written to FDA commissioner Robert Califf, HHS secretary Xavier Becerra, and National Institutes of Health (NIH) acting director Lawrence Tabak raising questions about a “revolving door” between FDA and major drug companies. The letter is based on a Project Veritas interview
Drugs
FDA says staff from CDER and CBER will be part of a new International Council on Harmonization (ICH) formal expert working group to develop the guideline (ICH M15), which will outline principles for Model-Informed Drug Development (MIDD). This comes on the heels of ICH’s 11/2022 finalized concept
Drugs
FDA researchers say the alternative tools used by the agency to evaluate data reliability and integrity, good clinical practice (GCP) compliance, and subject safety during the Covid-19 public health emergency were “critical in informing the agency’s regulatory decisions for marketing applications while mitigating the spread of Covid-19.