First Approved Drug for Fibrodysplasia Ossificans Progressiva in Japan: Sofonous®

Japan's Breakthrough Approval for Sofonous® in Treatment of FOP

On February 19, 2026, IPSEN Corporation announced a significant milestone in Japan with the approval of Sofonous® (Palovarotene) for treating Fibrodysplasia Ossificans Progressiva (FOP) by the Ministry of Health, Labour and Welfare. This is the first drug in Japan specifically authorized to help alleviate the symptoms of this rare and debilitating bone disease.

FOP is characterized by the abnormal formation of bone in muscles and soft tissues, leading to progressive loss of mobility and a significantly reduced life expectancy. Patients living with this condition can experience extraordinary challenges as their bodies develop unwanted bone formations that may restrict movement and function. It is estimated there are approximately 60 to 84 affected individuals within Japan and at least 900 worldwide, affecting their daily lives severely.

IPSEN's CEO, Seigo Noda, expressed his commitment to FOP patients and their families, stating, “We have continuously strived to provide treatment options for Japanese patients with FOP, who are in desperate need of solutions. We are truly delighted to have obtained the manufacturing and sales approval for this innovative drug that has shown to suppress heterotopic ossification, which has a serious impact on these patients' lives.”

Historically, the treatment options for FOP have been limited, mainly focusing on symptom management. With the approval of Sofonous®, patients aged eight and above, as well as those 10 years and older, can now access a medically validated option that addresses the root cause of the unwanted bone growth. This drug emerged from a multi-center, open-label Phase III MOVE trial, which presented critical data on its safety and efficacy.

The trial involved 107 patients who received oral Palovarotene, translating to about 12% of the estimated global FOP patient population. The results showed significant reductions in the volume of ectopic bone formation, marking a promising advancement compared to standard treatments. Notably, patients in the Palovarotene group experienced a 54% reduction in ectopic ossification volume on an annualized basis compared to the control group. The side effects reported were consistent with known safety profiles of systemic retinoids.
Understanding the severe implications of FOP on daily living, where many patients may find it increasingly difficult to care for themselves or engaging in tasks of daily living, the approval brings hope. Reports indicate that many affected by FOP require constant assistance by the age of 30, highlighting the urgency for effective treatment solutions.

The impact of this acknowledgment by the health authorities cannot be overstated. The question of how we can support individuals with FOP at every level is crucial; they urgently need relief from the burdens that this condition imposes.

IPSEN stands as a global biopharmaceutical company dedicated to delivering innovative medicines across oncology, rare diseases, and neuroscience. With nearly a century's experience in drug development and a commitment to advancing healthcare, IPSEN aims to reach over 100 countries, positively impacting patient lives.

In a world where everyday challenges can be compounded by such grievous disabilities, the approval of Sofonous® represents not just a new treatment avenue, but a beacon of hope for those diagnosed with Fibrodysplasia Ossificans Progressiva, bringing renewed possibilities for improved quality of life.

For more information about Sofonous® and its implications, you can visit IPSEN's official website.

For questions, please contact IPSEN at 03-6205-3483.