PARIS–(BUSINESS WIRE)– Regulatory News:
Ipsen (Euronext: IPN; ADR: IPSEY) today announced, following discussions with the U.S. Food and Drug Administration (FDA), that a partial clinical hold effective immediately, for the pediatric population under the age of 14 was issued for studies conducted under IND120181 and IND135403 evaluating the investigational drug candidate palovarotene for the chronic treatment of fibrodysplasia ossificans progressiva (FOP) and multiple osteochondromas (MO), respectively. The partial clinical hold applies to the pediatric population (patients under the age of 14 years) currently participating in the Phase 2 (PVO-1A-202/204 and PVO-2A-201) and Phase 3 (PVO-1A-301) studies in all clinical sites at global level. The FDA is allowing the studies to continue to treat patients 14 years of age and older.
The partial clinical hold was issued following recent safety reports submitted by the company to the FDA of cases of early growth plate closure in pediatric patients with FOP treated with palovarotene. The FDA has placed the studies on partial clinical hold pending review of additional details regarding these events and plans to issue additional requests for information within the next 30 days. Although no serious adverse events (SAEs) related to early growth plate closure in the MO study have been reported to date, this study has been included in this hold due to the occurrence of these events with chronic dosing in the FOP program. Since the MO study is a primarily pediatric study with the upper age of enrollment at 14 years, all subjects currently participating in the study will have interruption of treatment until further notice and no new patients will be enrolled while the partial clinical hold is in effect.
At Ipsen, the safety of patients is always a top priority, and the company is committed to ensuring the safe and effective use of its medicines. Ipsen is committed to researching and developing therapies for children and adults living with FOP and MO, two rare and devastating bone diseases with no current therapeutic treatment options.
Ipsen is committed to working diligently with the FDA to provide all requested information with the goal of resolving the partial clinical hold. Ipsen continues to prepare the FDA New Drug Application (NDA) filing for palovarotene in acute/flare-up FOP.
Ipsen is a global specialty-driven biopharmaceutical group focused on innovation and Specialty Care. The Group develops and commercializes innovative medicines in three key therapeutic areas – Oncology, Neuroscience and Rare Diseases. Its commitment to oncology is exemplified through its growing portfolio of key therapies for prostate cancer, neuroendocrine tumors, renal cell carcinoma and pancreatic cancer. Ipsen also has a well-established Consumer Healthcare business. With total sales over €2.2 billion in 2018, Ipsen sells more than 20 drugs in over 115 countries, with a direct commercial presence in more than 30 countries. Ipsen’s R&D is focused on its innovative and differentiated technological platforms located in the heart of the leading biotechnological and life sciences hubs (Paris-Saclay, France; Oxford, UK; Cambridge, US). The Group has about 5,800 employees worldwide. Ipsen is listed in Paris (Euronext: IPN) and in the United States through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information on Ipsen, visit www.ipsen.com.
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