Gene therapy for wet age-related macular degeneration is one of two later-stage gene therapies flagged in CVS Caremark’s latest pipeline report that are outliers because they target conditions afflicting millions of patients and could dramatically increase Part B spending.

US suitability petition submissions see an uptick against the backdrop of GDUFA III new goals for the FDA's response to such petitions. The Pink Sheet tracks some of the activity as applicants seek to tap market opportunities without the need for new clinical data.

The Duchenne muscular dystrophy treatment missed its primary endpoint of time to stand from supine in the RACER53 study, raising more questions about using dystrophin changes to support accelerated approval and the ability of functional endpoints to detect clinical change.

The Center for Drug Evaluation and Research’s novel approvals concentrated in August, November, and December, while January and June were the slowest months, according to a Pink Sheet analysis of 2014-2023 data.

EMA charges participants to receive scientific advice through the fledgling program, unlike the US FDA, which may not fit the budgets of some complex generic sponsors. At the same time, sponsors also may simply not be aware the program exists yet.

Acknowledging that it may be a barrier to some extent, EMA’s senior scientific specialist of clinical pharmacology Kevin Blake notes that EU reference medicinal products are a legal requirement for bioequivalence studies and cannot be circumvented.

While EU preparations are underway to introduce strict legal requirements for all AI systems, the UK has doubled down on its flexible, non-regulatory framework. In this second of a two-part article, a lawyer explains the pros and cons of each for pharma and medtech firms.

Flexible thinking and rigorous standards will both be needed to develop psychedelics as drug therapies in order to surmount the many complicating factors, from unique ‘set and setting’ aspects to functional unblinding, speakers at Reagan-Udall Foundation meeting agree.

FDA had recommended studying a twice-weekly dose, but Novo declined because it would add complexity to treatment for type 1 diabetes. However, the firm’s proposed strategy for reducing hypoglycemia risk with once-weekly insulin icodec would create its own complexity, panel said.

Patient-reported diabetes treatment satisfaction questionnaire used in Novo Nordisk’s ONWARDS 6 study had methodological shortcomings, US FDA said. Without reliable PRO data, benefits from a once-weekly injection remained theoretical, while hypoglycemia risks were real, panelists said.

The EU is on track to get its first vaccine for preventing the disease caused by chikungunya virus. Thirteen other products have also been recommended for pan-EU approval. 

England’s chief scientific officer explains how the NHS Genomic Medicine Service has led to faster access to targeted cancer drugs and outlines the organization’s ongoing work in pharmacogenomics, liquid biopsies and personalized RNA vaccines.

Gene therapy for wet age-related macular degeneration is one of two later-stage gene therapies flagged in CVS Caremark’s latest pipeline report that are outliers because they target conditions afflicting millions of patients and could dramatically increase Part B spending.

Sometimes even the US FDA’s regulatory flexibility may not be enough to approve rare disease treatments in very small populations, but agency officials worry that creating a new pathway could be problematic.

The US FDA does not seem ready to depart from its safety-focused emphasis on product characterization as cell therapies become more complex, despite calls for approaches to allow human studies that let different edits “compete” and “find the winners inside the human tumor.”

The European Medicines Agency is this week deciding whether the planned EU filings for sipavibart, lifileucel and Blenrep – which was previously approved but withdrawn in the EU and the US – merit an accelerated assessment.

US suitability petition submissions see an uptick against the backdrop of GDUFA III new goals for the FDA's response to such petitions. The Pink Sheet tracks some of the activity as applicants seek to tap market opportunities without the need for new clinical data.

The US FDA’s cancer drug review managers say most sponsors are happy to continue with virtual meetings, even though in-person opportunities are offered. Managers also see advantages to the virtual format for staff development.

CBER Director Peter Marks outlines a streamlined process to approval for treating different mutations of the same gene. NCATS’ Philip Brooks tells the Pink Sheet the approach avoids the need to “start from scratch for every new mutation.”

The Center for Clinical Trial Innovation was created to be CDER’s hub for expertise and information on innovative clinical trial approaches. C3TI lead Kevin Bugin talks to the Pink Sheet about the program's genesis and what it hopes to accomplish.