Saroglitazar for PBC Granted Orphan Drug Status in EU

In the European Union (EU), Orphan Drug designation is granted by the European Medicines Agency (EMA). The status is given to drugs or biologics intended to treat, prevent, or diagnose patients with rare, chronically debilitating, or life-threatening conditions. Within the EU, this status refers to conditions affecting no more than 5 in every 10,000 people. According to The Hindu BusinessLine, pharmaceutical company Zydus Cadila recently shared that its treatment Saroglitazar Mg, designed for patients with primary biliary cholangitis (PBC), received Orphan Drug designation from the EMA.


So what exactly is Saroglitazar? According to a study published in Hepatology, Saroglitazar is:

a dual peroxisome proliferator activated receptor-α/γ agonist.

In a Phase 2 clinical trial, researchers evaluated the safety, efficacy, and tolerability of Saroglitazar for patients with PBC. Ultimately, the results were promising, spurring the administration of the Orphan Drug status. Outside of Orphan Drug designation within the EU, Saroglitazar also received Orphan Drug and Fast Track designations within the United States.

As a result of its Orphan Drug status, Zydus Cadila also receives certain benefits from the EMA. For example, the company will have lower regulatory fees, 10 years of market exclusivity (once approved), and trial assistance.

Beyond PBC, Saroglitazar may also benefit patients with non-alcoholic fatty liver disease (NAFLD), nonalcoholic steatohepatitis (NASH), and diabetic dyslipidemia.

Primary Biliary Cholangitis (PBC)

Formerly called primary biliary cirrhosis, primary biliary cholangitis (PBC) is a chronic liver disease caused by an abnormal immune response. In patients with PBC, the immune system mistakenly attacks bile ducts, though doctors are not exactly sure why. Immunological disturbances, as well as environmental factors, are still under review. However, PBC is not caused by alcohol consumption. In patients with PBC, progressive liver damage stops bile from moving out of the liver. As bile builds up, it can cause scarring and, ultimately, liver failure. PBC tends to affect females more often than males. Additionally, the condition often affects those in middle age. Symptoms include:

  • Severe fatigue
  • Dry mouth
  • Persistent itchiness (pruritus), especially on the bottom of the feet
  • Darkening skin
  • Chronic diarrhea
  • Stomach and throat bleeding
  • Fragile bones
  • Muscle and joint pain
  • Small white bumps under the skin (often around the eyes)
  • Jaundice (yellowing of the skin and eyes)
  • Swelling of the lower extremities
  • High cholesterol
  • Unintended weight loss
  • Fluid buildup in the abdomen
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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