USA More than 40 years ago in 1983, the Orphan Drug Act was passed in the US to support and incentivise the development of rare disease therapies. Despite the over 6,000 orphan drug designations (ODD) granted by the FDA since then, it is estimated that only around five percent of rare…
USA The US rare and ultra-rare disease market constitutes a cornerstone of the worldwide pharmaceutical industry, representing a full 52.3 percent of the total global market share in revenue for that therapeutic area. America also plays an outsized role in driving international medical innovation in that field – housing nearly 60…
USA Matt Heck, founder and CEO of Sentynl Therapeutics, outlines the company’s acquisition-led rare disease strategy following its integration into Zydus Group in 2017. He discusses building a focused portfolio of life-extending paediatric therapies, leveraging orphan drug incentives, prioritising early diagnosis, expanding internationally, and partnering closely with patient foundations to maximise…
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