Expert Panel Approves Roche’s Orphan Drug For Blood Disorder, Waives Local Clinical Trials
Swiss pharma giant Roche has received approval from a panel of experts to sell a drug for a rare blood disorder, paroxysmal nocturnal hemoglobinuria (PNH), in India, waiving the requirement to conduct clinical trials on Indian soil, News18 has learnt.
The drug – a humanised complement inhibitor C5 monoclonal antibody – is indicated for the treatment of PNH which is a rare and life-threatening condition leading to the premature destruction of red blood cells. This disorder impacts red blood cells (responsible for transporting oxygen), white blood cells (which defend the body against infections), and platelets (crucial for blood clotting). PNH can develop at any age but generally, it is diagnosed among young adults. The condition leads to acute anaemia, fatigue, blood clots, and kidney disease.
According to an Indian study published in 2021, the incidence of PNH in the country among people with blood disorders is between 11.8% and 35.3% with a mean age of presentation of 34 years. In Europe, PNH has an incidence of 1 to 10 cases out of every 10 lakh people.
The drug, crovalimab, controls PNH via an injection every four weeks. One of the biggest advantages of crovalimab is that it can be self-administered just beneath the skin. In its global phase 3 clinical trial, the drug met co-primary efficacy endpoints of transfusion avoidance and control of haemolysis, or the ongoing destruction of red blood cells.
SEC told Roche to do post-marketing surveillance, phase 4 trials
The panel of experts has agreed to the company’s request for a local clinical trial waiver.
The committee noted that the drug is approved in 35 countries including the US, European Union, Japan, and others. The committee also noted that crovalimab is an “orphan drug” and proposed for the treatment of a “rare disease”.
“After detailed deliberation, the committee recommended for grant of permission to import and market the drug with a waiver of local Phase III clinical trial…” said the minutes of the meeting while adding certain conditions behind the approval.
According to the first and second conditions, the drug should be only prescribed by a haematologist for the treatment of haematological disorder PNH.
Also, the firm is now required to conduct a phase 4 study in India and, accordingly, Roche should submit a protocol for the phase 4 study within three months of grant of permission.
Phase 4 studies are also known as post-marketing surveillance where the company is expected to identify rare adverse reactions or harmful effects, assess efficacy, and optimise the drug’s use.
This phase is crucial for determining the future prescription of the drug, potential label expansions, and new indications that may emerge as more individuals begin using the drug in real-world settings rather than just in clinical trials.The SEC has asked the firm to modify the proposed indication in line with the US-FDA-approved indication.
Experts praise the move, call it patient-friendly
Experts believe this is a positive step, given that PNH is a rare condition with few available treatment options.
“Monoclonal antibodies are not available for this disease as yet in India. The only curative therapy with us is bone marrow transplant, which is a very aggressive treatment,” Dr Gaurav Dixit, unit head of haemato-oncology at Gurugram-based Artemis Hospital, told News18. “This drug would be a very good and non-toxic option for our PNH patients.”
According to an Indian study published in 2021, the incidence of PNH in the country among people with blood disorders is between 11.8% and 35.3% with a mean age of presentation of 34 years. In Europe, PNH has an incidence of 1 to 10 cases out of every 10 lakh people.
The drug, crovalimab, controls PNH via an injection every four weeks. One of the biggest advantages of crovalimab is that it can be self-administered just beneath the skin. In its global phase 3 clinical trial, the drug met co-primary efficacy endpoints of transfusion avoidance and control of haemolysis, or the ongoing destruction of red blood cells.
SEC told Roche to do post-marketing surveillance, phase 4 trials
The panel of experts has agreed to the company’s request for a local clinical trial waiver.
The committee noted that the drug is approved in 35 countries including the US, European Union, Japan, and others. The committee also noted that crovalimab is an “orphan drug” and proposed for the treatment of a “rare disease”.
“After detailed deliberation, the committee recommended for grant of permission to import and market the drug with a waiver of local Phase III clinical trial…” said the minutes of the meeting while adding certain conditions behind the approval.
According to the first and second conditions, the drug should be only prescribed by a haematologist for the treatment of haematological disorder PNH.
Also, the firm is now required to conduct a phase 4 study in India and, accordingly, Roche should submit a protocol for the phase 4 study within three months of grant of permission.
Phase 4 studies are also known as post-marketing surveillance where the company is expected to identify rare adverse reactions or harmful effects, assess efficacy, and optimise the drug’s use.
This phase is crucial for determining the future prescription of the drug, potential label expansions, and new indications that may emerge as more individuals begin using the drug in real-world settings rather than just in clinical trials.The SEC has asked the firm to modify the proposed indication in line with the US-FDA-approved indication.
Experts praise the move, call it patient-friendly
Experts believe this is a positive step, given that PNH is a rare condition with few available treatment options.
“Monoclonal antibodies are not available for this disease as yet in India. The only curative therapy with us is bone marrow transplant, which is a very aggressive treatment,” Dr Gaurav Dixit, unit head of haemato-oncology at Gurugram-based Artemis Hospital, told News18. “This drug would be a very good and non-toxic option for our PNH patients.”
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