CDSCO Panel Approves Akums' Proposal to Manufacture, Market Nitisinone Dispersible Tablets for Hereditary Tyrosinemia Type 1

New Delhi: The Subject Expert Committee (SEC), functioning under the Central Drugs Standard Control Organisation (CDSCO), has recommended granting permission to Akums Drugs and Pharmaceuticals to manufacture and market Nitisinone Dispersible Tablets in the strengths of 2 mg, 5 mg, and 10 mg.

This came after the firm presented their proposal for marketing and manufacturing of Nitisinone Dispersible Tablets 2mg/5mg/10mg along with justification for bioequivalence (BE) and clinical trial (CT) waiver.

The committee noted that hereditary tyrosinemia type 1 (HT-1) is a rare disease and the applied strengths are approved as Tablets.

Hereditary tyrosinemia type 1 (HT1) is a severe, inherited metabolic disorder caused by a deficiency of the enzyme fumarylacetoacetate hydrolase (FAH), which prevents the proper breakdown of the amino acid tyrosine. This leads to a buildup of toxic metabolites that damage the liver and kidneys, causing symptoms like poor growth, jaundice, and, if untreated, liver failure and increased risk of liver cancer.

The condition is managed with a low-tyrosine diet and the medication nitisinone, which, when started early, can significantly improve outcomes and survival rates.

Nitisinone is a competitive inhibitor of 4-hydroxyphenyl-pyruvate dioxygenase, an enzyme upstream of fumarylacetoacetate hydrolyase (FAH) in the tyrosine catabolic pathway. By inhibiting the normal catabolism of tyrosine in patients with hereditary tyrosinemia type 1 (HT-1), nitisinone prevents the accumulation of the catabolic intermediates maleylacetoacetate and fumarylacetoacetate.

At the recent SEC meeting, the expert panel reviewed the firm's request for BE and CT waivers in detail.

After detailed deliberation, the committee recommended for the grant of permission for the manufacture and marketing of Nitisinone Dispersible Tablets 2 mg/5 mg/10 mg for the proposed indication.