Stoneman Syndrome Treatment Market Size, Share, and Industry Analysis, By Drug Class (Corticosteroids, Non-steroidal anti-inflammatory drugs (NSAIDs), Retinoids, and Others), By Route of Administration (Oral and Parenteral), By Distribution Channel (Hospital Pharmacies, Retail Pharmacies, and Online Pharmacies), and Regional Forecast 2024-2032

Stoneman syndrome, also called Fibrodysplasia Ossificans Progressiva (FOP), is a rare genetic disorder marked by the abnormal replacement of muscle and connective tissue with bone, leading to the formation of bone outside the skeleton. This disease results in the musculoskeletal abnormalities and heterotopic ossification in both the developmental and postnatal stages. There are currently limited treatment options for this disease, which emphasizes the growing need for the development of innovative treatments. The drugs, such as corticosteroids, retinoids, skeletal muscle relaxants, and NSAIDs, are used to slow down the progression of this disease.

The market expansion is majorly driven by a rise in research and development activities, which offers numerous opportunities for novel drug development.

• For instance, in January 2020, Regeneron Pharmaceuticals, Inc., presented findings from the Phase-2 clinical trial LUMINA-1. This trial aimed to evaluate garetosmab (REGN2477) as a potential treatment for Fibrodysplasia Ossificans Progressiva in a double-blind, placebo-controlled manner.

Moreover, several key market players are undergoing strategic partnerships for the development and commercialization of new drugs in the market.

• For example, in October 2019, Clementia Pharmaceuticals, a subsidiary of Ipsen Biopharmaceuticals, Inc. and Blueprint Medicines Corporation, entered into an exclusive agreement to jointly develop and commercialize BLU-782. This highly selective investigational ALK2 inhibitor is targeted for the treatment of Fibrodysplasia Ossificans Progressiva.

Furthermore, the global Stoneman syndrome treatment market was slightly affected by the COVID-19 pandemic in 2020, mainly due to disruptions in healthcare services, clinical trials, and research. Patients with rare diseases such as Stoneman syndrome encountered difficulties in obtaining treatment and medical care amidst the pandemic. Moreover, the patients with this disease were at risk of decreased physical, mental, and dental health as a result of the COVID-19 pandemic.

Key Insights

The report covers the following key insights:

• Epidemiology of Stoneman Syndrome


• New Product Launches, By Key Players


• Key Industry Developments: Mergers, Acquisitions, and Partnerships


• Pipeline Analysis, By Key Players


• Impact of COVID-19 on the Stoneman Syndrome Treatment Market

Segmentation

Analysis by Drug Class

Based on drug class, the market can be segmented into corticosteroids, NSAIDs, retinoids, and others. The retinoids segment accounted for a significant proportion of the Stoneman syndrome treatment market in 2023. The increasing regulatory approvals for biologics to treat Stoneman syndrome is one of the key factors responsible for segment growth.

• For instance, in August 2023, Ipsen Biopharmaceuticals, Inc. announced the U.S. FDA approval of its drug, Sohonos (palovarotene), for the treatment of Stoneman syndrome. This drug is available in the form of capsules and it is the first and only drug approved for the treatment of Stoneman syndrome till now.

Moreover, various market players are engaged in clinical trials aimed at developing and launching effective drugs for treating Stoneman syndrome.

• For instance, in August 2020, Keros Therapeutics, Inc. announced the completion of a Phase-1 trial evaluating the efficacy of KER-047 in the treatment of a rare musculoskeletal disorder, Fibrodysplasia Ossificans Progressiva (Stoneman syndrome).

Regional Analysis

North America held a significant share of the global Stoneman syndrome treatment market in 2023 and is expected to maintain its position over the forecast period. The region's highest growth is mainly due to the presence of favorable healthcare infrastructure and the highest epidemiology of the disease across North America as compared to the other regions.

• For instance, as per data provided by the National Institutes of Health in August 2021, the documented worldwide occurrence of FOP, as per patient organization records, the confirmed cases of registered FOP patients range from about 0.65 per million in North America and 0.47 per million in Western Europe to around 0.27 per million in Latin America, 0.05 per million in Africa, and nearly 0.04 per million in the Asia Pacific region.

Moreover, some of the key industry players in the market are focusing on receiving the regulatory approvals to provide effective medications for the treatment of Stoneman syndrome. This is one of the factors contributing to the regional growth in the Stoneman syndrome treatment market.

• For instance, in August 2022, BioCryst Pharmaceuticals, Inc., announced that the U.S. FDA granted Orphan Drug Designation to its ALK-2 Inhibitor, BCX9250, for the treatment of Stoneman syndrome.

Increasing number of clinical trials and growing awareness about the disease in the North America region are some of the other factors positively impacting the market growth.

Key Players Covered

The report includes the profiles of stoneman syndrome treatment market key players such as Ipsen Biopharmaceuticals, Inc., BioCryst Pharmaceuticals, Inc., Blueprint Medicines Corporation, Keros Therapeutics, Inc., and Regeneron Pharmaceuticals Inc.

Key Industry Developments

• In November 2021, Aurobindo Pharma Limited received the U.S. FDA approval for its Prednisone Tablets USP, 1mg. This drug is used for the treatment of several bone and muscle-related disorders.


• In June 2020, Junya Toguchida laboratory announced that it had found an effective drug for the treatment of Fibrodysplasia Ossificans Progressiva.


• In November 2019, BioCryst Pharmaceuticals, Inc. initiated a Phase-1 clinical trial with BCX9250, an oral ALK-2 inhibitor for the treatment of the Stoneman syndrome. Furthermore, the company received the Orphan Drug Designation in August 2022 from the U.S. FDA for treating Stoneman syndrome.


• In April 2019, Ipsen Biopharmaceuticals, Inc. announced the acquisition of Clementia Pharmaceuticals. Ipsen closed this acquisition after receiving the U.S. FDA approval for the drug palovarotene for the treatment of Stoneman syndrome.