Orphans Should Live Alone
For larger organizations with interests in rare diseases, Bionest Partners believes it is necessary to maintain a separation from the rest of the company in order to keep the culture needed for successful product commercialization. Easier said than done.
You may also be interested in...
Hard-won experience from the first wave of broad-market biosimilars will inform development of "orphan biosimilars," but Navigant Consulting says that biopharmas entering this orphan market will face a unique set of challenges.
BioMarin’s licensing of the PARP inhibitor talazoparib to Medivation in August reduces BioMarin’s development expenses and helps it to meet its stated goal of being non-GAAP earnings positive in 2017. Medivation gets another compound it can develop in key oncology indications it is already pursuing with its core product, the prostate cancer drug Xtandi.
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.