RARE Daily

Pfizer Shelves Two Rare Disease Programs

January 31, 2023

Rare Daily Staff

Pfizer said it will discontinue to rare disease programs in the clinic, as it decided to discontinue eight programs in its pipeline across all indications.

Pfizer, which had been bolstered by sales COVID vaccines, is bracing for about a 30 percent fall in revenue and a 50 percent drop in earnings per share in 2023, leading it to adjust its organizational structure and thin its pipeline.

Among a number of programs the company said it discontinued from development since November 1, which ranged from COVID-19 infection to various cancers, the company said it ceased development of recifercept for achondroplasia and PF-06755347 for chronic inflammatory demyelinating polyneuropathy and primary Immune thrombocytopenia

In 2019, Pfizer acquired Swiss biotech Therachon, which gave it a potential first-in-class therapy for achondroplasia.

Achondroplasia is the most common type of short-limbed dwarfism and occurs in one in 15,000 to 40,000 newborns worldwide, or about 250,000 people. It is caused by mutations in the FGFR3 gene that controls making a protein involved in the development and maintenance of bone and brain tissue, leading to dwarfism of the arms and legs, limited range of motion at the elbows, large head size, small fingers, and more serious spinal problems. Treatment usually involves medication with growth hormone.

Therachon’s experimental, soluble recombinant human fibroblast growth factor receptor 3 (FGFR3) decoy, a mechanism of action that is believed to normalize the overactive FGFR3 signaling pathways that underlie bone development abnormalities associated with achondroplasia. Pfizer was in phase 2 development of the therapy.

PF-06755347 is an experimental therapy that Pfizer licenses from Gliknik in 2013 for $25 million in additional to undisclosed milestone payments and royalties. Pfizer paid an additional $15 million milestone when it moved the experimental therapy into the clinic for chronic inflammatory demyelinating polyneuropathy (CIDP), a rare neurological disorder.

PF-06755347 is a recombinant immunomodulation candidate that was designed with the hope of meeting or exceeding the efficacy of the blood product immune globulin (IVIg) in treating autoimmune diseases.

Last month, Barron’s reported that Pfizer was reducing its efforts in early-stage rare disease research including the development of viral-based gene therapies. The company still has a rare disease pipeline with a dozen candidate extending from phase 1 to registration.

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