Country: BRAZIL | Region: SOUTH AMERICA | Type: Drug Approval | Keywords: #advancedtherapyproducts #anvisa #clinicaltrials #drugregistration #orphandrugs #rarediseases #regulation
PRICENTRIC BRIEF:
- In 2019, Brazil’s National Health Surveillance Agency (ANVISA) approved 21 orphan drugs and 30 clinical trials, a record for the regulator
- The recent approval of Resolution of the Collegiate Board (RDC) 338/2020, which takes effect June 1, 2020, addresses the registration of advanced therapy products and the rules for conducting clinical trials with such investigational medicines, now providing Brazil with the necessary regulatory bases to develop and register high technology products based on human cells and genes
- Some notable drugs approved by ANVISA in 2019 in orphan indications include Vitrakvi (in two dosage forms), Libtayo, Tegsedi, Besponsa, and Takhzyro
THE DETAILS
BRASILIA, Brazil – In 2019, Brazil’s National Health Surveillance Agency (ANVISA) approved 21 orphan drugs and 30 clinical trials, a record for the regulator.
About 13 million Brazilians have rare diseases, according to the Ministry of Health data.
Oftentimes, advanced therapy products have the potential greatly benefit patients with rare diseases for whom no medical alternatives are available. This includes cell and gene therapies.
The recent approval of Resolution of the Collegiate Board (RDC) 338/2020, which takes effect June 1, 2020, addresses the registration of advanced therapy products and the rules for conducting clinical trials with such investigational medicines.
With this resolution, Brazil now has the necessary regulatory bases to develop and register high technology products based on human cells and genes, thereby becoming a reference in Latin America.
The 21 medicines approved for rare diseases in 2019 are:
- Millidock Medical and Pharma Products’ Translarna (ataluren)
- United Medical’s Cresemba (isavuconazonium sulfate)*
- Pharmedic’s Nitikabs (nitisinone)
- Bayer’s Vitrakvi (larotrectinib)*
- Recordati’s Carbaglu (carglumic acid)
- Iclusig (ponatinib hydrochloride)
- Evobrig (brigatinib)
- Tegsedi (inotersen)
- Erfandel (ardafitinib)
- Novartis’ Piqray (alpelisibe)
- Galafold (migalastat hydrochloride)
- Bayer’s Nubeqa (darolutamide)
- Zodiac’s Defitelio (defibrotide)
- Uno Healthcare’s Crysvita (burosumab)
- Pfizer’s Besponsa (inotuzumab ozogamicin)
- Sanofi’s Libtayo (cemiplimab)
- Shire’s Takhzyro (lanadelumab)
- Roche’s RoPolivy (polatuzumab vedotin)
- Crystadane (anhydrous betaine)
*For Cresemba and Vitrakvi, two dosage forms were considered, which according to ANVISA, means two products were approved.
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