SpringWorks Therapeutics
NEWS
SpringWorks Therapeutics sprung out of Pfizer’s storeroom, when a rare disease advocacy group pushed to keep a program for neurofibromatosis alive. This method could work for “every rare disease under the sun,” advocates say.
SpringWorks Therapeutics is the perfect case study for rescuing a discontinued assets. It’s time to repeat the process for every rare disease, experts say.
Following an initial report from Reuters, Merck KGaA confirmed that it is in talks with SpringWorks for a potential acquisition, though details of its offer have yet to be revealed.
The rare disease space is awaiting two FDA verdicts in February, one for a genetic disease and another for a non-malignant tumor.
Patient advocacy groups aided in the development of the very first marketed drugs for certain rare diseases, including progeria and Friedreich’s ataxia.
The Ogsiveo tablets are the first approval therapy for the rare subtype of soft tissue sarcomas, which can lead to severe pain and disability and previously were treated primarily through surgery.
The first half of this year saw milestone approvals for rare disease therapies, and the FDA has several more such decisions on its calendar in the second half of 2023.
SpringWorks, Eli Lilly, EMD Serono and Mirati released data ahead of the European Society of Medical Oncology (ESMO) 2022 Meeting in Paris.
SpringWorks announced the expansion of its partnership with GlaxoSmithKline to develop nirogacestat in combination with GSK’s Blenrep for relapsed or refractory multiple myeloma.
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