RARE Daily

Ipsen Plans to Move Forward with Filing for FOP Drug

August 25, 2020

Rare Daily Staff

Ipsen said an analysis of data from its late-stage study of palovarotene in the rare bone disorder fibrodysplasia ossificans progressive showed it reduced mean annualized abnormal growth of bone in non-skeletal tissue in pediatric and adult participants with FOP compared to untreated patients from a natural history study over 24 months.

The analysis comes after the company had paused the study because futility criteria had been met in a pre-specified interim analysis.

Results from the MOVE trial demonstrated a 62 percent reduction in mean annualized new heterotopic ossificiation (HO) volume (abnormal bone growth in soft tissue) in participants treated with palovarotene.

FOP is an ultra-rare, genetic disorder that affects approximately 1.36 per million individuals worldwide and is characterized by formation of bone in soft and connective tissues, known as HO. Sporadic episodes of painful soft tissue swelling, called “flare-ups,” can precede HO. HO is permanent and leads to severe functional limitations in joint mobility, progressive and cumulative disability, and to shortened life expectancy. There are currently no approved treatments for the reduction or prevention of heterotopic bone formation in FOP.

“Accumulation of HO across the body is the defining characteristic of FOP and severely limits physical function over time,” said Robert Pignolo, Division of Geriatric Medicine and Gerontology, Department of Internal Medicine, Mayo Clinic. “The MOVE trial provides important insight into long-awaited treatment strategies and demonstrates that the oral therapy palovarotene can reduce new HO volume, representing an important therapeutic option in FOP especially in older children and adults.”

“We are pleased to present the third interim analysis results of the phase 3 MOVE trial in FOP, evaluating Ipsen’s oral investigational therapy palovarotene, to the global community at the ASBMR annual meeting,” said Jim Roach. “Based on preliminary discussions with the FDA, Ipsen’s intent is to move forward with an NDA submission and discussions with the EMA are ongoing. We remain committed to working with regulators and are delighted to be one step closer to our goal of bringing this potential treatment option to people living with FOP in a timely manner.”

Photo: Robert Pignolo, Division of Geriatric Medicine and Gerontology, Department of Internal Medicine, Mayo Clinic

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