Market Analysis: Global Centronuclear Myopathies Drug Market
Market Definition: Global Centronuclear Myopathies Drug Market
Centronuclear Myopathy is a rare genetic disease which primarily causes muscle weakness. The protein myotubularin which is essential for our ability to breathe and swallow is either missing or dysfunctional due to a mutation to the MTM1 gene. This protein is essential for our muscles to contract. Many patients with centronuclear myopathy need immediate intervention and need around-the-clock care including ventilator support in a feeding tube.
According to the statistics published in the Valerion Therapeutics, it was estimated the overall prevalence of this disease is 1: 50,000 male births. Huge financial support from the government and increase in strategic alliances between the companies are key factors for market growth.
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Family history of centronuclear myopathy is driving the market growth
Increase special designation from the regulatory authorities is boosting the market growth
High specific demand of novel treatment is accelerating the market growth
Huge financial support to the researchers for developing novel intervention is enhancing the market growth
Limited availability of medicinal treatment options due to low prevalence of centronuclear myopathy is restraining the market growth
Fewer approvals of drugs from the regulatory authorities is hindering the market growth
Lack of trained personnel and stringent safety regulations is hampering the market growth
Segmentation: Global Centronuclear Myopathies Drug Market
Autosomal Centronuclear Myopathies
By Treatment Type
Night Time Ventilation
By End Users
By Distribution Channel
Rest of South America
Rest of Europe
Rest of Asia Pacific
Middle East & Africa
Rest of Middle East & Africa
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Key Developments in the Market:
In August 2019, Dynacure in a collaboation with Ionis Pharmaceuticals, Inc received Orphan Drug designation from the FDA for the treatment of Centronuclear Myopathies. With the Orphan Drug designation, companies can obtain financial incentives from the government along with 7-year period marketing exclusivity.
In November 2017, Ionis Pharmaceuticals, Inc. out-licensed worldwide development and commercialization right to Dyanacure for IONIS-DNM2-2.5Rx (Dyn101), a generation 2.5 antisense drug targeting dynamin 2 receptor for the treatment of centronuclear myopathy (CNM). Under the deal terms, Dyanacure paid USD 5.00 million as a licensee fee and eligible to pay additional cash of nearly USD 205.00 million as milestone payment. Additionally, Ionis Pharmaceuticals, Inc. is entitled to receive royalties on future net sales of the product. This collaboration provides potential treatment for patients suffering from centronuclear myopathy.
Key Market Players:
Few of the major competitors currently working in the global centronuclear myopathies drug market are Audentes Therapeutics, Ionis Pharmaceuticals, Inc, Dynacure, Valerion Therapeutics, Biophytis, ARMGO Pharma Inc, Stealth BioTherapeutics Inc and others.
Demand Side: Doctors, Surgeons, Medical Consultants, Nurses, Hospital Buyers, Group Purchasing Organizations, Associations, Insurers, Medical Payers, Healthcare Authorities, Universities, Technological Writers, Scientists, Promoters, and Investors among others.
Supply Side: Product Managers, Marketing Managers, C-Level Executives, Distributors, Market Intelligence, and Regulatory Affairs Managers among others.
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