Causal biology & big data, ultrarare drugs at FDA, and more: a BioCentury podcast

Identifying causal biology in an age of big data — where to start? Plus: latest in Stealth’s bid to get ultrarare drug through FDA, and Lundbeck buys Longboard

There is a growing mandate among researchers and VCs to provide proof of causal human biology for new targets. On the latest BioCentury This Week podcast, BioCentury’s editors discuss the different strategies being deployed to identify causal links to disease using observational patient data or human cell models, including the challenges that come with each approach and the various computational methodologies companies are using.

They also discuss the outcome of FDA’s advisory committee meeting on Barth syndrome candidate elamipretide from Stealth Biotherapeutics Corp., and the implications of the discussion for review of ultrarare disease therapies more broadly.

Diving into the deal of the day, the editors review the proposal by H. Lundbeck A/S (CSE:HLUN B) to acquire Longboard Pharmaceuticals Inc. (NASDAQ:LBPH) for $2.6 billion, and discuss how the biotech’s therapy for developmental epilepsies may stack up against competitors.

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