Home HEALTH India Launches First Locally Manufactured Drugs for Rare Diseases

India Launches First Locally Manufactured Drugs for Rare Diseases

The Indian medical devices sector is playing a pivotal role in shaping the narrative of 'Make in India' on the global stage. The adoption of advanced medical technologies is enhancing the efficiency of healthcare delivery, ensuring that even remote areas have access to state-of-the-art diagnostic and treatment options

In a groundbreaking development, the Union Health Ministry of India has announced the launch of the first four domestically produced drugs for rare diseases, also known as orphan diseases. This significant move is poised to dramatically reduce medication costs, making treatment more accessible to individuals grappling with these conditions.


 Until now, drugs used for treating rare diseases were primarily imported, rendering them prohibitively expensive. This situation often compelled individuals to resort to crowdfunding to afford treatment.


The newly launched generic medications are tailored for the treatment of four rare conditions: Wilson’s disease, Gaucher’s disease, Tyrosinemia-Type 1, and Dravet-Lennox Gastaut syndrome.


 The introduction of locally manufactured drugs is expected to significantly bring down costs, making these medications accessible at a fraction of their previous market prices. Patients can now acquire these drugs for 60 to 100 times less than the previous costs.


 The Health Ministry is actively working to make drugs available for additional rare diseases, including Phenylketonuria and Hyperammonemia. Approvals for these drugs are awaited, signaling a broader initiative to enhance accessibility and affordability. The Central Government has prioritized 13 rare diseases and sickle cell disease. In addition to the ongoing efforts, a sickle cell disease drug formulation tailored for children will be made available. The decision to manufacture these drugs locally followed extensive discussions with academia, pharmaceutical industries, organizations, and the CDSCO department of pharmaceuticals.


Officials from the Health Ministry believe that the launch of these drugs is a “game-changer” and will bring about a paradigm shift in the landscape of rare disease treatment not only in India but also in other low-income nations.


 The World Health Organization defines rare diseases as health conditions with particularly low prevalence, affecting less than 1 per 1,000 individuals. Despite their rarity, collectively, these diseases impact about 6-8 percent of the population in any country.

The launch of locally manufactured drugs for rare diseases marks a historic milestone, reflecting India’s commitment to making vital treatments more accessible and affordable. This initiative is poised to bring relief to individuals grappling with rare conditions and sets a precedent for transformative changes in healthcare accessibility.



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