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Brazil has become the first among the Latin American countries to approve the marketing of gene therapy products. Specifically, the Brazilian Health Regulatory Agency (ANVISA) recently granted marketing authorization for Novartis’ gene therapy products Luxturna® and Zolgensma®.  As published in the Brazilian Federal Register, Luxturna® received its marketing authorization on August 6th and a few days later, on August 17th, Zolgensma® received its marketing authorization. Prior to ANVISA’s final decision, both products had been approved by the Brazilian Technical Commission of Biosafety (CTNBio), which is responsible for evaluating biosafety of Genetically Modified Organisms (GMOs) in Brazil.

Luxturna®

Luxturna® (voretigene neparvovec) is indicated for the treatment of adult and pediatric (children over 12 months) patients with vision loss caused by bi-allelic retinal pigment epithelium (RPE) mutation-associated hereditary retinal dystrophies.  In Brazil, no other therapeutic option available for the treatment of this rare disease until approval of Novartis’ gene therapy.

It took 232 business days from the submission of the marketing authorization application for ANVISA to provide its decision. As part of the administrative approval procedure, ANVISA and Novartis signed a Term of Commitment to ensure that the company will conduct additional trials with Brazilian patients to continue to monitor the safety and effectiveness of Luxturna®  on a long-term basis.

Zolgensma®

Zolgensma® (onasemnogene abeparvovec) is indicated for the treatment of pediatric patients less than 2 years of age with type 1 spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron (SMN1) gene and with bi-allelic mutations in the SMN1 gene and up to 3 copies of the SMN2 gene.

After 149 business days of analysis, ANVISA concluded that the product’s benefits surpassed its risks and granted the marketing authorization. However, because it is a new gene therapy, the authorization was granted on a exceptional basis, meaning that Novartis is required to send data on Zolgensma’s® ongoing trials as agreed to in the signed Term of Commitment.

Novartis also commited to conduct additional studies with Brazilian patients to enable the evaluation of whether Zolgensma® maintains a positive benefit-risk balance.  Additionally, Novartis also agreed to train (i) healthcare professionals who will perform infusions of the drug; and (ii) Brazilian hospitals in the appropriate storage and handling of  Zolgensma®.

Regulatory framework and next steps

The Brazilian regulatory framework on advanced therapeutic products is very new.  At the end of 2018, ANVISA’s Collegiate Board of Directors approved Rule #260/2018, which defines the technical standards for conducting clinical trials with experimental advanced therapy products.  On February 26, 2020, ANVISA’s Rule #338/2020 was published which provides the requirements for obtaining a marketing authorization for advanced therapy products. The regulation classifies advanced therapy products into three different categories: (i) advanced cell therapy; (ii) gene therapy; and (iii) tissue engineering. Both Luxturna® and Zolgensma® fall into the second category, gene therapy.

The next step for Novartis to introduce Luxturna® and Zolgensma® into the Brazilian market involves receiving price approval from the Brazilian Drugs Market Regulation Chamber (CMED).  This approval is expected by the end of 2020.  There is much anticipation regarding the pricing of these drugs, particularly since Zolgensma® is currently considered to be the world’s most expensive drug (at USD $2.125 million).

The approval of the first gene therapy products in Brazil also brings up the issue of whether these drugs will become available in the Public Healthcare System (SUS). The analysis of whether a drug is incorporated into the SUS is conducted by the National Commission for Incorporation of Health Technologies (CONITEC), which issues a recommendation to the Ministry of Health. While Luxturna® and Zolgensma® are currently not in the SUS, the Brazilian population can obtain free access to these products through a court order.

Although there are still some a few steps to be taken until Luxturna® and Zolgensma® truly get into the hands of patients in Brazil, the approval itself and the consolidation of a regulatory framework on advanced therapeutic products is expected to promote the development of this pharmaceutical in Brazil.

This post was written by Lisa Mueller, Roberto Rodrigues, Ana Calil and Natalia Toledo of Licks Attorneys.

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