Drug maker Cadila Healthcare Limited (Zydus Cadila) has informed that it has received ‘Orphan Drug Designation’ (ODD) from the EU regulator, European Medicines Agency (EMA), for Saroglitazar Mg to treat patients with Primary Biliary Cholangitis (PBC).

Earlier, the US drug regulator US Food and Drug Administration (USFDA) had granted ‘Orphan Drug Designation’ and ‘Fast Track Designation’ to Saroglitazar Mg for PBC.

ODD status in Europe is given to medicines with the potential to be safe and effective treatments for rare, life-threatening or chronically debilitating conditions affecting no more than 5 people in 10,000 people.

PBC is a liver disease, caused due to progressive destruction of the bile ducts in the liver, which leads to reduction of bile flow – a condition referred to as cholestasis.

Progressive cholestasis

PBC can lead to progressive cholestasis and even turn fatal. And with an increasing number of people being affected by it, there is a pressing need to develop therapies, which help achieve an adequate reduction in Alkaline Phosphotase (ALP) or bilirubin, reduce strong side effects of existing drugs such as pruritus or increase in LDL-c, and bring in better tolerance and efficacy.

For Zydus, this development will open new prospects, as it provides companies with a range of incentives, including assistance with trial protocols, reduced regulatory fees, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralised marketing authorisation procedure valid in all EU Member States, and a 10-year period of market exclusivity if the treatment eventually is approved.

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The company informed that Saroglitazar Mg is a potent and selective peroxisome proliferator-activated receptor alpha and gamma dual agonist.

The results of Phase 2, prospective multicentre randomised double-blind, placebo-controlled study to evaluate the safety, tolerability and efficacy of Saroglitazar Mg in patients with PCB was presented earlier at the annual meeting of the American Association for the Study of Liver Diseases, it informed.

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Speaking on the development, Pankaj R Patel, Chairman, Zydus Group, said: “We are pleased that the EMA has granted an Orphan Drug Designation to Saroglitazar Mg for the treatment of PBC. This is a serious health condition and we are committed in our clinical development efforts to improve the quality of life of patients suffering from PBC with a safe and efficacious treatment.”

The treatment options are still evolving for PBC, and Saroglitazar holds immense potential based on its safety and efficacy profile so far.