AstraZeneca Pharma India has received approval from the Central Drug Standard Control Organisation (CDSCO) to import and market Eculizumab concentrate for solution for infusion 300 mg, targeting Atypical Hemolytic Uremic Syndrome (aHUS). This approval, however, is contingent upon AstraZeneca conducting a Phase IV clinical trial to establish the drug’s safety and effectiveness within the Indian population.
The approval follows AstraZeneca’s presentation of global clinical trial data, along with a request to waive a local clinical trial. Despite this, the Subject Expert Committee (SEC) emphasized the necessity for a Phase IV trial due to the unmet medical need for aHUS treatment in India.
Atypical Hemolytic Uremic Syndrome (aHUS) is a rare genetic disorder that causes small blood clots to form in blood vessels, leading to serious health issues such as kidney failure and heart disease. Eculizumab, a recombinant humanized monoclonal antibody, works by inhibiting complement protein C5, thereby preventing the formation of the terminal complement complex C5b-9 and reducing complement-mediated damage.
The SEC’s approval includes several conditions:
- AstraZeneca must conduct a Phase IV clinical trial to confirm the drug’s safety and efficacy in India.
- The proposed indication should match the U.S. Food and Drug Administration (USFDA) approved indication, with modifications submitted to CDSCO.
- The drug should be prescribed for aHUS based on recommendations from Centres of Excellence (CoE) institutions.
AstraZeneca is required to submit the Phase IV trial protocol to CDSCO within three months of receiving marketing authorization for review by the committee.