Booming Growth in Centronuclear Myopathies Drug Market Analysis and Forecast to 2026| Audentes Therapeutics, Ionis Pharmaceuticals, Inc, Dynacure, Valerion Therapeutics, Biophytis

Market Analysis: Global Centronuclear Myopathies Drug Market

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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Market Drivers

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


Market Restraints

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

By Types

Myotubular Myopathy

Autosomal Centronuclear Myopathies

Others


By Treatment Type

Night Time Ventilation

Gastrostomy

Physiotherapy

Antibiotics

Others


By Diagnosis

Muscle Biopsy

Molecular Testing

Others


By End Users

Hospitals

Homecare

Specialty Clinics

Others


By Distribution Channel

Hospital Pharmacy

Online Pharmacy

Retailers

Others


By Geography

North America

U.S.

Canada

Mexico


South America

Brazil

Rest of South America


Europe

Germany

France

United Kingdom

Italy

Spain

Russia

Turkey

Belgium

Netherlands

Switzerland

Rest of Europe


Asia-Pacific

Japan

China

South Korea

India

Australia

Singapore

Thailand

Malaysia

Indonesia

Philippines

Rest of Asia Pacific


Middle East & Africa

South 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.

Competitive Analysis:

Global centronuclear myopathies drug market

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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.

Primary Respondents

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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