Rare Disease Report

Pfizer Creates New Smaller Company to Focus on Rare Diseases

SEPTEMBER 26, 2017
James Radke
Big Pharma company Pfizer Inc. is taking a page out of some of the smaller biotech companies that it would normally acquire. Pfizer is creating a smaller company to develop orphan drugs.

Today, Pfizer announced plans for SpringWorks Therapeutics, which will be focused on just 4 experimental drugs, 3 of which are for rare diseases. The new creation will use the flexibility of its smaller size to develop the drugs.

According to Pfizer, SpringWorks will connect scientists, biopharmaceutical partners, patient groups, funders and philanthropists to develop the drugs.

Initially, SpringWorks will focus on drugs for 3 rare conditions; desmoid tumor, neurofibromatosis, and hereditary xerocytosis, as well a drug to manage post-traumatic stress disorder.

Desmoid tumors are a slow-growing, benign tumors that grow in connective tissue and affects 900 to 1,200 people each year. There is no approved therapy for these tumors and in most cases, the tumors are removed surgically. Usually, the tumors reappear after surgery. While not cancerous, these tumors can be painful and depending on their location, can be very disruptive to a person’s quality of life.

Neurofibromatosis consists of benign tumors surrounding neurons. There are numerous types of neurofibromatosis with the most common, neurofibromatosis type 1, generally involves nerves near the skin and the most noticeable symptom is abnormal pigmentation (cafe-au-lait spots) and/or freckling in atypical locations (armpits, groin). Neurofibromatosis type 2 usually involves tumors affecting the inner ear.

Hereditary xerocytosis is a rare form of anemia in which the person’s red blood cells are ‘leaky’ and allow salt and water to enter the cells and burst. There is no treatment currently available for this condition.

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