The story of the rare disease market in the last few years has been centered around growth. Although many rare disease drug developers have traditionally been mom-and-pop companies, large pharmas are starting to see more promise in the space as deals tick up.
In the last month, Biogen acquired the rare disease drugmaker Reata Pharmaceuticals, which received FDA approval for its Friedreich’s ataxia drug Skyclarys in March, for $7.3 billion, and AstraZeneca’s Alexion deepened its rare disease investment in a deal to buy Pfizer’s early-stage gene therapy portfolio for up to $1 billion.
Earlier this year Novartis also snapped up the rare kidney disorder-focused Chinook Therapeutics for $3.2 billion. And, in the biggest biopharma deal of 2022, Amgen purchased Horizon Therapeutics for $27.8 billion in December to expand its rare disease pipeline.
Amid the flurry of deals and growing interest in the space, approvals for new rare disease drugs have skyrocketed. Between 2015 and 2022, rare disease drugs accounted for half of all FDA approvals.
Of course, countless challenges still remain — from clinical trial recruiting woes, to shifting regulatory landscapes and complexities with incentive structures.
For insight into how leaders are navigating these and other obstacles, join us on Wednesday as we sit down with some of the industry’s leading rare disease experts to talk clinical trials, regulations and more.
And in the meantime, check out our rare disease coverage to brush up on some of the hot-button trends and hurdles in the space.