Approval Standards
Sponsors making “big statements” with “no evidence” to back them up is one of the biggest issues faced by the UK drug regulator when it is assessing the regulatory dossiers for advanced therapy medicinal products, a quality assessor from the Medicines and Healthcare products Regulatory Agency says.
The US FDA will seek industry feedback on the Commissioner’s National Priority Voucher pilot during a June public meeting as the ultra-fast drug review program marks its first year amid growing scrutiny of its scope, timelines, and sponsor obligations.
Context of use, human biological relevance, technical characterization and fitness for purpose are the key elements in validating novel methods to replace animal testing, the FDA said in a new draft guidance.
The US FDA’s Rare Disease Hub is becoming more concrete, with dedicated 2026 funding and momentum for a more substantial user-fee allocation going forward.
Significant viral drift and a temporarily rebuilt committee set the stage for a high‑stakes VRBPAC vote on updates to the three influenza vaccine strains for the 2026–2027 season.
The FDA’s contrasting decisions on leucovorin and idebenone reveal a tightening regulatory stance where only therapies showing strong mechanistic rationale and large, credible treatment effects can overcome the limitations of nontraditional evidence sources.
Instead of requiring a three-way pharmacokinetic bridging study that compares the US reference product, foreign reference product and proposed biosimilar, a sponsor should justify the relevance of clinical data comparing a proposed biosimilar to a foreign product.
Vinay Prasad will leave the FDA less than a year after his initial arrival, having served two tenures as head of the biologics center.
The agency requested uniQure test AMT-130 against a control arm in which patients undergo a sham procedure. The treatment may be more akin to other neurodegenerative gene therapies than the Huntington's chorea drugs that were approved based on placebo-controlled trials.
Albert Bourla once again publicly criticized CBER Director Vinay Prasad’s attempts to overrule career FDA staff decisions on vaccines, but was more positive about other US policy reforms.
While defending the FDA’s decision-making on rare disease therapies, along with Director Vinay Prasad, US FDA Commissioner Martin Makary talked about review issues in ways commissioners usually do not.
Neonatal drug development struggles with the lack of a standard of care and pervasive off‑label use, but the FDA’s growing preparedness for AI and real‑world evidence approaches offers an opening to harness NICU data to accelerate neonatal‑focused therapies.
During a Senate hearing, rare disease stakeholders said the FDA is not engaging with patients as much as before and suggested it may be hindering drug development.
EU regulators must strive to balance scientific rigor with “common sense” when it comes to advanced therapy medicinal products, particularly for patients with no other treatment options, Immatics’ chief medical officer tells the Pink Sheet.
From enforcing new GMP norms to risk-based inspections and 850 CAPA notices, India’s drugs regulator is moving to tighten compliance across domestic pharma units. A striking step up in the regulator’s internal scientific cadre and more regulatory rationalization are also on the cards.
The Pink Sheet's list of EU centralized approvals of new active substances has been updated to include five new products, one of which is Aumseqa (aumolertinib), Hansoh Pharma's treatment for non-small cell lung cancer.
Pink Sheet editors consider the impact of Trump Administration-aligned proposals on the FDA user fee program, as well as discuss the latest updates on the Moderna flu vaccine application.
The third complete response letter (CRL) from the US Food and Drug Administration for Outlook Therapeutics’ wet age-related macular degeneration (wAMD) drug is the latest instance of the agency requiring more confirmatory evidence than prior expectations for an eye disease treatment.
Clinical trials are just one of many ways to assess credibility, FDA Commissioner Martin Makary and CBER Director Vinay Prasad said in a NEJM article that described replacing the traditional standard of two clinical trials with a new default expectation of one study.
The US FDA commissioner also defended CBER Director Vinay Prasad’s role in the agency’s decision to refuse to file the company’s application for its novel flu vaccine.