India’s First Medicine to Treat Duchenne Muscular Dystrophy Cleared for Clinical Trials

After 13 years of research, India’s first personalized medicine for Duchenne muscular dystrophy (DMD) has received the green signal from the Drug Controller General of India (DCGI), paving the way for clinical trials.

The DCGI has not provided details of the compound it has approved, but noted that what is approved in the USA has been indigenously made in India.

In the USA, there are two types of antisense therapies for DMD: eteplirsen (Exondys 51 from Sarepta Therapeutics [Nasdaq: SRPT]) and drisapersen, from BioMarin Pharmaceutical (Nasdaq: BMRN), with only eteplirsen approved for marketing.

DMD is a rare genetic disorder that has affected 0.8 million male children in India. The disease usually affects male children between the ages of three and 15. Patients tend to die by the age of 20.

The current treatment available in the country is prohibitively expensive. The available therapy in the global market is called antisense oligonucleotide (AON), developed by US and European pharmaceutical companies.

AON therapy ranges between $409,566 to $546,088 per year for the treatment of a single child. The drug is thus beyond the reach of most Indian patients and their families.

It was this one fact that motivated a team of Indian scientists to develop a chemical formula to treat DMD, in a manner that is effective as well as affordable.

The initial assessment of the Indian drug indicates a pricing of about $20,491 annually, which is expected to go down further with bulk production.

Led by a team of researchers from the Indian Association for the Cultivation of Science, Kolkata, and professors from the Department of School of Applied and Interdisciplinary Sciences, the Dystrophy Annihilation Research Trust (DART) bagged the nod from the DCGI and CDSCO, as well as permission from the Ministry of Health and Family Welfare to initiate human clinical trials.

Trials are scheduled early 2021. DART is planning to conduct extensive multi center clinical trials, across India in nine different hospitals.

Since the modus operandi of Indian scientists is based on targeting the wrong genetic sequence, the same technique also can be used for the treatment of diseases such as cancer, thalassaemia and diabetes. Researchers are focussing on cancer.

Source: ThePharmaletter