FDA
In this episode of Denatured, you’ll hear from Jerome Barley, commercial director at Avion Medical and Ryan Klein, vice president, business development, US and APAC at Resolian. We discuss why Australia has become a magnet for early clinical development, and whether its speed, regulatory clarity and tax incentives are pushing it toward a more integrated biotech role.
The FDA has outlined a process for allowing a single application to cover multiple variants of a gene therapy. At the same time, the Centers for Medicare & Medicaid Services has revealed plans to expand the Cell and Gene Therapy Access Model to increase patient access to these products.
Drug developers increasingly have the option of seeking regulatory approval and commercializing a medicine themselves, rather than accepting an early exit offer. Doing so requires a worldwide strategy.
CROs cite faster startup timelines, regulatory predictability and tax incentives as reasons for growing interest in Australia. State investments in manufacturing, translational research and advanced therapies aim to expand that advantage into a broader biotech ecosystem. The question remains whether Australia can support drug development at broader scale.
AbbVie is eyeing regulatory filings in 2027 for the skin disease drug lutikizumab after clearing multiple endpoints in a late-stage trial, but the results have disappointed some analysts, raising questions about its commercial prospects.
Troriluzole, in development for spinocerebellar ataxia, was rejected nearly a year ago when the FDA took issue with the design of Biohaven’s registrational study. The agency has now agreed to review a new application and render a decision in the first quarter of 2027.
In this episode of Denatured, you’ll hear from Bruce Culleton, CEO at ProKidney and Demetri Maxim, CEO at Nephrogen. We discuss why kidney disease has long been a hard target for cell and gene therapies, and what new delivery strategies, therapeutic approaches and regulatory clarity mean for commercially viable products.
Hibsago’s approval—a functional cure for certain patients with a hepatitis B infection—further validates Ionis Pharmaceuticals’ antisense platform, which has already generated two other new drug approvals this year.
Following several recent initiatives, the FDA’s newest rare disease framework sets out to standardize the agency’s approach to scientific evidence and bring rare disease therapies to patients faster.
Data released by the FDA show that Revolution Medicine’s newly approved pancreatic cancer drug underperformed in a large subgroup of patients, but analysts say the biotech still has a clear winner.
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