"Across pharma and academia, an estimated 5,000+ shelved drug candidates were discontinued for reasons unrelated to safety or efficacy. Each represents a potential therapy for conditions that, in many cases, have no approved treatment at all. Industry stakeholders have a unique opportunity to collaborate on identifying these compounds. Aligning these assets with capable and motivated partners will benefit both drug developers and patients."
"No company can commit the resources to develop every promising drug, especially those designed for very small patient populations. It takes an extraordinary amount of time and money to bring a drug to market - And it's risky. Even after entering clinical trials, nine in 10 candidate medicines fail. Companies must prioritize candidates that fit their strategy and offer the strongest return."
"Of the roughly 7,000 identified rare and neglected diseases with known molecular causes, only about 500 have an approved treatment. Families facing those diagnoses can't wait decades for something new to be invented."
Pharmaceutical companies and academic institutions have discontinued over 5,000 drug candidates for reasons unrelated to safety or efficacy. These shelved compounds represent potential therapies for rare and neglected diseases, many of which currently lack approved treatments. The Gomekli case demonstrates this opportunity: Pfizer's abandoned cancer drug was licensed to SpringWorks Therapeutics through the Children's Tumor Foundation, resulting in FDA approval and a $3.4 billion acquisition by Merck. Companies cannot develop every promising drug due to resource constraints and financial risk, particularly for small patient populations. Of approximately 7,000 rare diseases with known molecular causes, only 500 have approved treatments. Strategic collaboration between pharmaceutical companies, biotech startups, and patient advocacy organizations can unlock these dormant assets for patients with limited treatment options.
#rare-diseases #drug-repurposing #pharmaceutical-partnerships #shelved-compounds #biotech-innovation
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