The Success Story of Mirdametinib: A Lesson in Rescuing Shelved Pharma Assets
Once destined for Pfizer’s R&D junk pile, the neurofibromatosis treatment mirdametinib has now emerged as a success story in drug development. Initially shelved due to AstraZeneca’s lead in the market with selumetinib, mirdametinib has now been approved by the FDA under the brand name Gomekli for both adults and children with neurofibromatosis type 1 with plexiform neurofibromas.
Turning the Tide
CEO of the Children’s Tumor Foundation, Annette Bakker, saw the potential in mirdametinib and convinced Pfizer to spin out SpringWorks Therapeutics to further develop the drug. This decision ultimately led to FDA approval, marking a significant milestone for neurofibromatosis patients and highlighting the importance of reevaluating shelved pharma assets.
Rescuing Shelved Assets
Drug development often faces high failure rates, with many candidates being scrapped for non-scientific reasons. Bakker’s success with mirdametinib emphasizes the need to rescue shelved assets that hold value for patients and have the potential for commercial success.
The Role of Nonprofits
Bakker believes that nonprofits play a crucial role in identifying and developing shelved assets that could benefit specific patient populations. By working with companies and facilitating drug development partnerships, nonprofits like the Children’s Tumor Foundation can help bring valuable drugs to market.
Barriers and Incentives
Despite the promise of rescuing shelved assets, there are significant barriers in the industry. Bakker advocates for transparent reporting systems between companies and nonprofits to facilitate partnerships and ensure financial compensation for their contributions.
Creating a Partnership
By incentivizing pharma companies to reevaluate and develop shelved assets, a public-private partnership can be formed to prevent valuable drugs from languishing unused. The success of mirdametinib serves as a testament to the potential impact of such collaborations in the pharmaceutical industry.
Overall, the story of mirdametinib highlights the importance of giving shelved assets a second chance and underscores the valuable role that nonprofits can play in bringing these drugs to market for the benefit of patients worldwide.
