From a Crinetics Pharmaceutical press release: Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical-stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, announced that the U.S. Food and Drug Administration (FDA) has granted paltusotine Orphan Drug Designation for the treatment of acromegaly.
The FDA’s Orphan Drug Designation program provides orphan status to drugs defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan Drug Designation qualifies the sponsor of the drug for certain development incentives, that may include exemption from FDA prescription drug user fees, financial incentives for qualified clinical development, and seven years of market exclusivity in the U.S. if the treatment is approved.
“The orphan drug designation by the FDA for paltusotine to treat acromegaly validates the encouraging clinical data we have seen so far in the Phase 2 ACROBAT Edge clinical trial, and underscores the need for a once-daily oral treatment option for patients suffering from this rare disease,” said Michael Monahan, Senior Director of Regulatory Affairs at Crinetics.
If you’d like to learn more about drug development and the regulatory challenges associated with bringing new drugs to market, listen to Michael Monahan in this informative podcast – Laws, guidelines, and best practices in new drug development – where he discusses the intricacies of the regulatory environment. Michael’s podcasts is one in a series about new drug development produced by PWN.
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