Pfizer is stepping back from early-stage rare disease R&D. After reviewing its portfolio, the Big Pharma has begun “exploring externalization opportunities for a number of highly innovative, niche programs” as part of a rethink of its approach to orphan indications.
Like many of its peers, Pfizer identified the rare disease space, and technologies such as gene therapies that unlock orphan indications, as a growth market and expanded through acquisitions and investment in internal projects. Late Thursday, financial publication Barron's revealed a shift in Pfizer’s approach, reporting a pullback from new viral-based gene therapies and early-stage rare disease work in general.
Pfizer confirmed the change in strategy in an emailed statement to Fierce Biotech. A spokesperson said the Big Pharma is “increasing earlier external innovation efforts where it makes sense to seek a greater balance between internal and external innovation,” leading to changes at the rare disease group.
“We are refocusing our approach to early discovery and development research in rare disease and oncology that involves embedding and continuing certain programs while exploring externalization opportunities for a number of highly innovative, niche programs to maximize the success for these potential medicines," they said.
“This approach will allow us to focus on programs where our innovation and investments are differentiated and where we are best positioned to generate high-impact medicines and vaccines. Depending on the externalization approaches taken, Pfizer may maintain ties to some of these programs through strategic investments,” the spokesperson added.
Pfizer has a track record of moving programs out of its pipeline, and off its list of costs, while maintaining a chance to profit if they deliver successful drugs. Exactly which drug candidates are on the block is unclear. Pfizer will continue to develop viral vector-based gene therapies for hemophilia A, hemophilia B and Duchenne muscular dystrophy, according to Barron’s, while shifting its investment to other delivery vehicles such as lipid nanoparticles (LNPs). Pfizer has LNP capabilities from its COVID-19 vaccines.