Research & Development Pfizer Steps Back From Early-Stage Rare Disease R&D

Pfizer Steps Back From Early-Stage Rare Disease R&D


Pfizer is withdrawing from research and development for rare diseases in their initial phases. As part of a rethinking of its strategy toward orphan indications, Big Pharma has begun examining the externalization prospects for a variety of extremely inventive, specialty products.

Pfizer, like many of its competitors, saw the rare disease market as appealing, and the technology associated with it, such as gene treatments that unlock orphan indications, as potential growth areas. Financial news outlet Barron’s reported late Thursday that Pfizer is shifting its focus away from novel viral-based gene treatments and early-stage rare disease efforts in general.

A Big Pharma representative stated that the company is shifting its focus from internal to external innovation early in cases where it makes sense to do so, such as with the rare disease division.

Among these programs are the viral vector gene therapies for hemophilia A and B as well as Duchenne muscular dystrophy. Pfizer’s hemophilia A and Duchenne muscular dystrophy initiatives are both in late-stage studies, with findings due within the next two years, while a gene treatment for hemophilia B has proven it can control bleeding in a late-stage trial.

The representative stated, “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.”

The spokesperson continued by saying that this course of action will allow the company to focus on programs best suited to its strengths, allowing it to develop medicines and vaccinations that would really make a difference for people in the long run. Pfizer may keep strategic investments tied to some of these programs, depending on the externalization methods used.

According to Barron’s, Pfizer will sell or license some of the oncology assets it is developing at an R&D center focused on Array BioPharma, which the company acquired for $11.4 billion four years ago. Through the first nine months of 2022, Pfizer made $285 million in sales of the targeted cancer medications Braftovi and Mektovi, both of which were acquired as part of the deal.

Moreover, Pfizer could increase its acquisition spending. The company has spent hundreds of billions of dollars over the past four years acquiring firms with authorized treatments like BioHaven Pharmaceutical, Global Blood Therapeutics and Array, as well as companies with therapies still in development like Reviral, Arena Pharmaceuticals and Trillium Therapeutics.

Companies often resort to externalization as a means of reducing research and development expenses or disposing of low-profit items. Over the past decade, AstraZeneca served as a case study for the pharmaceutical industry as it refocused from gastrointestinal and mental health drugs to oncology and respiratory treatments.

However, Pfizer finds itself in a different situation as a company.  Some of its most valuable assets in infectious disease and cancer are still expanding, and the company is still reaping record-breaking profits from sales of its COVID-19 vaccine and antiviral medicine. It is getting ready for a wave of launches brought on by its ongoing research and development activities, which might total 19 over the course of the next 18 months.

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