Pfizer Tafamidis to be sold on prescription of cardiologist only: CDSCO panel
New Delhi: Granting the permission of local clinical trial waver, the Subject Expert Committee (SEC) of the Central Drugs Standard Control Organization (CDSCO) has given its nod to pharmaceutical major Pfizer to import and market the Tafamidis Soft Gelatin Capsule 61 mg for transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
However, the approval came with the condition that the firm should conduct an active surveillance study and, accordingly, the firm should submit a protocol to CDSCO for review by the committee.
The expert panel further mandated the prescription of a cardiologists to sell the said drug.
This came in the wake of the proposal presented by pharmaceutical major Pfizer for the import and marketing of Tafamidis Soft Gelatin Capsules 61 mg along with the safety and efficacy data, regulatory approval status etc. in other countries before the committee.
Tafamidis is indicated to treat cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis in adults. Vyndaqel, which is tafamidis meglumine, and Vyndamax, which is tafamidis, were authorised by the FDA in May 2019 for the treatment of Transthyretin Amyloid Cardiomyopathy (ATTR-CM).
In general, Amyloidosis is a disease caused by a buildup of abnormal, "misfolded" proteins in the body called amyloid. Amyloid can be deposited throughout the body's tissues, which can cause organ damage.
In the case of Transthyretin amyloidosis (ATTR), the transthyretin protein "misfolds" or changes its shape in an abnormal way, and forms into fibrous clumps. Depending on the specific type of TTR amyloidosis, the amyloid is deposited in the heart, as well as in nerves and other organs, which can lead to permanent damage and organ malfunction and a heart disease called transthyretin amyloid cardiomyopathy (ATTR-CM).
Tafamidis is a selective stabilizer of transthyretin (TTR). Tafamidis binds to TTR at the thyroxine binding sites, stabilizing the tetramer and slowing dissociation into monomers, the rate-limiting step in the amyloidogenic process.
At the 88th SEC meeting for Cardiology and Renal held on 15.07.2021, 16.07.2021 and 19.07.2021 at CDSCO, the committee thoroughly examined Pfizer's proposal for the import and marketing of Tafamidis Soft Gelatin Capsule 61 mg.
The committee observed that:
1. Wild type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM) is a rare cardiovascular disease with no specific treatment.
2. There is an unmet need for selective stabilizer for both wild type TTR and amyloidgenic variants of TTR agents in the country.
3. The drug is approved in the USA, EU, Japan, Korea, Australia etc.
After detailed deliberation, the committee recommended the grant of permission to import and market the drug with a local clinical trial waiver for the proposed indication subject to the condition that the firm should conduct an active surveillance study and, accordingly, the firm should submit a protocol to CDSCO for review by the committee.
In addition to this, the committee further directed that the drug should be sold at retail under the prescription of cardiologists only.
