The Orphan Drug Boom: Gold Rush Or Flash In The Pan?
Rare disease drug development has ballooned in just three years, powered by the success of independent biotechs like Alexion, Big Pharma’s entrée into the field, and pressure from regulators and payors that is dis-incentivizing development of traditional primary care drugs. The positive momentum has intensified dealmaking in the orphan drug space and is giving investors confidence to back rare disease-focused start-ups. Some have coined the resulting movement the “orphan drug bubble,” but interest isn’t likely to deflate soon – at least not as long as Big Pharma continues to invest in the area and industry is able to sustain a favorable reimbursement climate for ultra-premium-priced drugs.
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Rivipansel, partnered with GlycoMimetics, is one of the most advanced drugs in Pfizer’s rare disease pipeline. Phase III studies have been delayed, however, due to a manufacturing issue impacting supply.
The global pharmaceutical industry is facing decreasing margins triggered by various internal and external reasons. To address the crisis effectively long term, we see pharma’s integration into care management, combined with data generation and analysis of real-world outcomes, as a way to charge for the value pharma adds.
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