With no domestic manufacturers developing drugs for people ailing with rare genetic diseases in India which is home to 8 crore such patients, the Government is working on a strategy to indigenously manufacture the drugs or bring generic drugs which are off- patent here as well as develop process chemistry for drugs under patent to make it affordable once patent expires.
Presently drugs are very costly and many times out of the reach of the patients too.
A senior official from the Department of Science and Technology (DST) under the Union Science and Technology has sought proposals from interested research groups for treatment of the rare genetic diseases.
“One-fifth of the global population is in India. High-genetic disease prevalence has been noted in affected communities with high genetic relatedness between parents and the majority of rare diseases in India occur at birth due to recessive genetic defects. While many of the commonly occurring rare diseases have viable therapies, most of these therapeutic agents are patent protected and very expensive for the Indian user.
“Thus, there is a large unmet need for indigenously manufactured therapeutic agents for rare diseases. Currently, a few pharmaceutical companies are manufacturing drugs for rare diseases globally and there are no domestic manufacturers in India except for Food for Special Medical Purposes (FSMP) for small molecule inborn errors of metabolism.
The DST has sought proposals from the interested pharma manufacturers as to “to bring generic drugs which are off- patent or to develop process chemistry for drugs under patent to make it affordable once patent expires,” said the official. The last date for submitting applications is July 15, the official informed.
According to the official, the rare diseases which may be targeted for the proposals include Lysosomal storage disorders, Gaucher disease, Mucopolysaccharidoses (all types), Pompe disease, Fabry disease, Niemann Pick disease, Metachromatic Leukodstrophy, Krabbe disease, Small molecule diseases (Phenylketonuria, Maple Syrup Urine Disease (MSUD), Neuromuscular Disorders such as Spinal Muscular Atrophy (SMA) and Dystrophinopathies
There are 7,000 different rare diseases known today, but many more still to be identified. Eighty-five to 90 percent of these are considered “serious or life threatening,” and for many, there are no known effective treatments – less than 10 percent of the known rare diseases have an approved treatment available. About 80 percent of rare diseases are caused by genetic abnormalities and half of these diseases impact children. Manufacturing of drugs for these categories of diseases are full of challenges.
According to the Tufts Center for the Study of Drug Development, it takes nearly four years longer to develop an orphan drug compared with medicines to treat more common conditions. Additionally, only about 6 percent of those drugs reach approval, compared with a 12 percent overall
