Drugs
Pharmaceutical Research and Manufacturers of America (PhRMA) says it believes the recommendations in an FDA draft guidance on considerations for developing chimeric antigen receptor (CAR) T cell products “are essential in helping sponsors navigate development issues in this rapidly evolving field.” In a 6/14 comment letter, PhRMA says that
Drugs
CDER researchers say their landscape analysis of the application of artificial intelligence (AI) and machine learning (ML) in drug regulatory submissions from 2016 to 2021 confirms an earlier prediction that AI/ML would play an increasingly important role in drug development. Writing in Clinical Pharmacology & Therapeutics, the authors say
Drugs
Despite all the challenges the Covid-19 pandemic has thrown at FDA, the agency’s drug Center has still been able to meet its user fee goal dates, and all domestic NDA pre-approval inspections that had been earlier delayed due to the pandemic have now been completed, CDER director
Drugs
FDA has approved a Rhythm Pharmaceuticals supplemental NDA for Imcivree (setmelanotide) injection, indicated for chronic weight management in adult and pediatric patients six years of age and older with obesity due to Bardet-Biedl Syndrome (BBS). The therapy was originally approved 11/2020 for chronic weight management in adult and
Drugs
CDER director Patrizia Cavazzoni says the work of the generic drug program during the Covid-19 pandemic is a prime example of how well CDER operated in difficult circumstances. In a memo to staff, Cavazzoni says, “Through cross-office collaboration, the program has been using new approaches during the pandemic
Drugs
Members of FDA’s Psychopharmacologic Drugs Advisory Committee will meet on 6/17 to consider Acadia Pharmaceutical’s resubmitted sNDA for Nuplazid (pimavanserin) for an indication to treat hallucinations and delusions associated with Alzheimer’s disease psychosis. The agency issued a briefing document in advance of the meeting indicating committee
Drugs
Four Alston & Bird attorneys say there is bipartisan support in both the House and Senate for the PDUFA 7 reauthorization legislation. Writing in a Health Care Legislative and Public Policy Advisory, the attorneys review some of the key provisions of the legislation. They report that PDUFA 7 places greater
Drugs
Pfizer says that as a result of a very low rate of death or hospitalization observed in standard risk Covid-19 patients, it will stop enrolling patients into the EPIC-SR trial of Paxlovid (nirmatrelvir [PF-07231332] tablets and ritonavir tablets). A statement says the company will include available data
Drugs
CDER director Patrizia Cavazzoni is personally spearheading CDER’s new rare disease efforts under its recently announced Accelerating Rare disease Cures (ARC) program, which is designed to speed and increase the development of effective and safe treatment options addressing the unmet needs of patients with rare diseases. Delivering a Center
Drugs
The American Medical Association (AMA) is encouraging FDA to approve over-the-counter (OTC) access to oral contraceptives without an age restriction. A statement issued during the AMA annual meeting says an FDA regulatory pathway exists for converting oral contraceptives from prescription products to OTC products and a required manufacturer
Drugs
An 8/4-8/20/21 FDA inspection at ACRC Studies, Poway, CA, conducted by Dr. Smitha Reddy, found “objectionable conditions” in the conduct of two clinical investigations. A 5/27 Warning Letter says the inspection was part of the agency’s Bioresearch Monitoring Program. The letter says a review
Drugs
A 7/27-9/3/21 FDA inspection at Hybrid Pharma, a Deerfield Beach, FL, outsourcing facility, determined that drug products produced there failed to meet the conditions of Section 503B of the Federal Food, Drug, and Cosmetic Act necessary for drugs produced by an outsourcing facility to be exempt
Drugs
FDA has approved an Alnylam Pharmaceuticals NDA for Amvuttra (vutrisiran), an RNAi therapeutic administered by subcutaneous injection once every three months for treating polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Approval was based on nine-month data from the HELIOS-A Phase 3 study that showed the therapy significantly
Drugs
Three Troutman Pepper attorneys say a 4/14 FDA draft guidance, Diversity Plans to Improve Enrollment of Participants from Underrepresented Racial and Ethnic Populations in Clinical Trials, is the agency’s first attempt to articulate detailed expectations for submission of a plan by trial sponsors to ensure that clinical trial
Drugs
FDA presiding officer Celia Witten says she expects an FDA advisory committee hearing on whether agency approval for Covis’ Makena (hydroxyprogesterone caproate injection) should be withdrawn will be held 10/17-10/19. The hearing dates are included in a letter to attorneys for CDER and Covis providing the questions
Drugs
A Brookings Institution white paper says there are steps FDA could take to better promote generic competition. The paper focuses on three areas of FDA authority that Brookings says “could be refined to better promote generic competition: the Citizen Petition mechanism, the approval of so-called complex generic drugs, and
FDA Policy/General
FDA commissioner Robert Califf does not think a total revamp of the drug accelerated approval program is needed in response to criticism that followed a number of drug indications being withdrawn because companies were unable to generate confirmatory effectiveness data. Delivering the commissioner’s address at the Food and Drug
Drugs
FDA and the Organization of Economic Cooperation and Development are launching an initiative to encourage the wide-scale adoption of a “whole-of-governments” approach to combating illicit medical products. A joint announcement says the initiative started with a 5/24 non-public meeting that focused on a successful whole-
Drugs
FDA has published a draft International Council for Harmonization (ICH) guidance, Quality Risk Management Q9 (R1), to provide a systematic approach to quality risk management for better, more informed, and timely decisions. The ICH says the document “serves as a foundation or resource document that is independent of, yet supports,
Drugs
A February inspection at Rentschler Biopharma’s German drug substance manufacturing plant has resulted in a nine-item FDA-483 that cites significant GMP violations. For example, FDA said the fill process is not controlled to an established specification. It also found the firm’s laboratory procedures for testing to
Drugs
Health Canada has given Amylyx Pharmaceuticals its first worldwide approval for Albrioza (sodium phenylbutyrate and ursodoxicoltaurine), with conditions, for treating amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease. “Clinical data demonstrated a statistically significant and clinically meaningful benefit in functional outcomes for people with ALS taking Albrioza
Drugs
FDA has removed a clinical hold on Homology Medicines and its pheNIX gene therapy clinical trial evaluating HMI-102 in adults with phenylketonuria (PKU). Homology says it is planning to inform clinical trial sites about changes to the protocol, which include a new steroid-sparing immunosuppression regimen that incorporates a