Approval Standards
The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include two new products, one of which is Vyjuvek, Krystal Biotech's advanced therapy for treating dystrophic epidermolysis bullosa.
The European Medicines Agency’s chief medical officer Steffen Thistrup argued that patients are able to understand the “complex regulatory and scientific issues” that the EMA committees discuss when they assess drugs.
The lack of a formal analysis of efficacy in castration-resistant prostate cancer patients without HRR mutations doomed Pfizer’s Talzenna at the US FDA's Oncologic Drugs Advisory Committee, as FDA oncology chief Richard Pazdur says chance cannot be ruled out.
The use of J&J’s Darzalex Faspro in high-risk smoldering multiple myeloma could result in overtreatment, but the FDA committee voted in favor of the new claim for delay in development of multiple myeloma.
The FDA may have violated laws and regulations along with many norms in how it released its planned changes for COVID-19 vaccine approvals.
The Oncologic Drugs Advisory Committee will vote on the risk-benefit of UGN-102 for low-grade, intermediate-risk, non-muscle invasive bladder cancer based on data from a single-arm trial, but the FDA also seeks input on requiring randomized studies for future treatments.
A US Supreme Court ruling in favor of the agency in an e-cigarette case has implications for drugs and other medical products as the justices decided not to disturb the FDA’s ‘change in position’ authority, while also creating a complex new landscape.
New CBER Director Prasad will "unleash a massive" framework on vaccines, FDA Commissioner Marty Makary said 15 May, raising questions about the impact on products nearing approval.
With an FDA review ongoing, Kennedy’s admission, made during a Senate hearing on the Health and Human Services Department’s budget request, suggests political officials could supersede scientific decisions.
The FDA continues to wait for a decision on whether its priority guidances will be published as HHS asks the public for potential regulation cuts to fulfill President Trump’s executive order.
Sanofi’s Xenpozyme and Sentynyl’s Nulibry are the first two case studies the FDA is using to continue educating rare disease sponsors on best practices.
Pink Sheet editors discuss the appointment of Vinay Prasad as the new director of the Center for Biologics Evaluation and Research and revelations that FDA layoffs now are hindering development of guidance documents.
HHS Secretary Robert F. Kennedy Jr. regularly decries the “conflicts of interest” he believes abound in advisory committees, but his concerns, as well as a recent “policy directive” eliminating industry representatives, seem driven by a misunderstanding of the panels’ jobs.
Cuts to the FDA’s policy and legal personnel have prevented dozens of product-specific guidances for generic drug development from being published.
Industry is concerned that Prasad may make regulatory flexibility tougher to obtain for cell and gene therapy, while vaccine and public health advocates are angry about Prasad’s criticisms of US COVID-19 policies.
Sponsors reported faster development times for products that joined the pilot program intended to speed rare disease treatments in CBER.
During its first quarter earnings call, Moderna avoided criticizing vaccine policy changes, but de-emphasized its flu/COVID-19 vaccine for those under age 50 and prioritized cancer programs.
CEO Len Schleifer said during the company’s first quarter earnings call that most of the complete response letters the company has received related to third-party suppliers, not efficacy or safety.
Three weeks after its user fee goal date, the FDA demanded a postmarketing commitment “to generate additional data,” Novavax said.
PhRMA questioned whether proof-of-concept studies are needed before an INTERACT meeting and said a draft guidance describing the timing of definitive toxicology studies creates confusion.