QR Group analyzes the impact of the arrival of Elevidys in Brazil and the advancement of gene therapy regulation
In July 2024, the National Health Surveillance Agency (ANVISA) approved Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy indicated to treat Duchenne muscular dystrophy (DMD) in Brazil. The drug is manufactured by Sarepta Therapeutics and had already received approval in the United States in 2023.
QR Group, a consulting firm specializing in health product regulation, analyzes the impacts of this approval from a regulatory, scientific and marketing perspective, especially with regard to the entry of innovative gene therapies in the country.
What is Elevidys and why is its approval relevant?
Elevidys is an innovative gene therapy indicated for children with DMD between 4 and 5 years of age who have a confirmed mutation in the dystrophin gene. The disease, of genetic origin, is progressive, rare and without a definitive cure – which makes the therapy a milestone in treatment.
The approval by ANVISA is based on clinical data that demonstrated functional improvement and stabilization of the motor condition in patients in this age group. The decision represents an important advance in personalized medicine and a sign that Brazil is aligned with global innovations in health.
Advances and challenges in the regulation of gene therapies in Brazil
The entry of Elevidys reinforces ANVISA’s ability to analyze and approve highly complex drugs, such as gene therapies. However, the regulatory process for this type of technology requires:
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Robust clinical dossiers, with a focus on long-term safety;
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Extended pharmacovigilance protocols;
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Technical capacity of the sponsoring companies to deal with the complexity of the technology;
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Alignment with international frameworks, such as those of the FDA and EMA.
The regulation of gene therapies is still maturing in Brazil, but the approval of Elevidys signals a promising path for the arrival of new genetic solutions in the country.
QR Group’s role in the entry of advanced technologies into the Brazilian market
Companies that develop gene therapies or intend to register highly complex products in Brazil face significant technical and regulatory challenges. QR Group offers:
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Strategic consulting for the registration of biotechnological and advanced products;
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Technical support in the construction of clinical and non-clinical dossiers;
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Direct relationship with ANVISA’s technical areas for regulatory clarifications;
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Risk mapping and definition of regulatory routes compatible with Brazilian requirements.
With international operations and in-depth knowledge of the national regulatory scenario, QR Group facilitates the safe and fast entry of innovative therapies into the Brazilian market.
Talk to QR Group
Does your company develop gene therapies or innovative technologies in health?
QR Group is your strategic partner to ensure registration and compliance with ANVISA requirements.
Source: QR Group