New Resources Could Improve Drug Development for Fibrodysplasia Ossificans Progressiva

Ipsen has just announced that they will be acquiring Clementia Pharmaceuticals. This merging of companies is exciting for the rare community because it will strengthen the resources, knowledge-base, and overall capabilities of Ipsen in developing and commercializing new treatments.

One of the first benefits this collaboration could have is an accelerated development of palovarotene for the treatment of multiple osteochondromas (MO) and Fibrodysplasia Ossificans Progressiva (FOP). MO is a chronic condition resulting in the formation of benign tumors in the bones. It results in limb deformity and restricted movement. The only current treatment option for patients is surgery, with some patients necessitating an upwards of 30 before they even reach adulthood. MO affects around 150,000 people around the world. FOP is caused by bone formations outside of the normal skeleton structure. It is progressive and can impede movement and shorten life expectancy. It impacts approximately 9,000 individuals around the world and there are no approved treatments currently available.

Both Ipsen and Clementia both pride themselves in their patient-centered approach and their focus on patient populations with high unmet needs. They expect their integration will lead to an elevated level of expertise, talent, and overall ability to aid in the advancement of therapeutic options for the rare disease community.

About The Companies

Ipsen focuses on oncology, rare disease, and neuroscience. Their portfolio includes therapies for renal cell carcinoma, neuroendocrine tumors, pancreatic cancer, and prostate cancer in addition to MO and FOP. They are a global company which has around 5,700 employees across the world. They sell drugs in over 115 different countries.

Clementia’s focus is ultra-rare bone disorders. Their lead drug candidate is palovarotene.

Palovarotene

Palovarotene is a retinoic acid receptor gamma (RARy) agonist. It works by inhibiting BMP signaling. The signaling of this protein is associated with the progression of both MO and FOP.

Both of these rare conditions are currently lacking any treatment options.

Palovarotene has already received Rare Pediatric Disease Designation, Breakthrough Therapy Designation, and Fast Track Designation by the FDA for FOP. Additionally, it has been given Orphan Drug Designation for MO and FOP by the FDA and EMA.

Current Trials

Palovarotene is currently in a Phase 3 trial for the treatment of FOP. It is also in a Phase 2 trial, which is evaluating the drug as a treatment for MO. Lastly, there is a Phase 1 trial investigating the drug as a therapy for dry eye disease. In addition to these trials, the therapy is being investigated for other rare conditions.

The goal is to submit a New Drug Application to the FDA for episodic flare-up of FOP by the second half of 2019. If the application is approved, palovarotene should launch commercially by the middle of 2020.

Ipsen expects to have fully acquired Clementia by the second quarter of this year. You can read more about this new integration here. Hopefully, we will see the benefits from this combination of minds and resources aid the rare community very soon.


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