doi:10.1038/nindia.2016.61 Published online 15 May 2016
India now has its first ever indigenously developed biological drug containing human stem cells.
A Bengaluru-based stem cell company, Stempeutics Research has received regulatory approval for 'limited marketing' of its product for the treatment of Critical Limb Ischemia (CLI) due to Buerger’s disease — the first stem cell based biologics to be approved by the Drugs Controller General of India (DCGI).
CLI is a progressive form of peripheral arterial disease which blocks the arteries in the legs resulting in reduced blood flow. This leads to severe pain and ulcers or necrosis which finally may require amputation. CLI affects 3.4 million patients in India and approximately 2 per 10,000 persons in the European Community & USA.
"Now there is hope for these patients," says Balu Manohar, the company's CEO. Its flagship product "stempeucel" — patented in the US, Japan, China and Malaysia — is derived from allogeneic pooled mesenchymal stromal cells (MSC), extracted from the bone marrow of adult voluntary donors.
"This is the first allogeneic MSC transplantation study in India," Manohar said adding that the pooling technology developed is also novel and has been patented.
The DCGI approval for "stempeucel" is based on clinical data generated in phase-1 and phase-2 trials on 90 subjects with Buerger’s disease. "In both Phase studies, significant improvement in blood flow is observed compared to placebo or control group of patient," Manohar said.
Although it is too early to say if Stempeucel would improve amputation-free survival in these patients, the clinical data suggests that disease progression has been halted and clinical improvement observed in several parameters, he said. "This improvement is consistent for a period of 6 months follow up of these patients."
Under DCGI's limited approval, Stempeutics can manufacture and sell its product to 200 patients on a cost recovery basis (limited to Rupees 150000 or $2200) per patient and submit the data to DCGI’s office before seeking full marketing authorization. "They have given us a time window of two years," Manohar said. "Our goal is to complete the study on 200 patients and seek full marketing authorisation by the end of next year."
"This is a huge win for patients, for Stempeutics, and for the field of MSC therapy," Jeffrey Karp, Associate Professor of Medicine at Harvard Medical School who is also in the company's advisory board told Nature India. "Also, we must commend the Indian regulators for promoting regenerative medicine for major unmet medical needs."
Stempeutics is working to globalise its product, which has already been granted the Advanced Therapy Medicinal Product and Orphan Drug Designation status by European Medicinal Agency. "This will help us fast-track commercialisation in Europe," said Manohar.