US biotech Alnylam Pharmaceuticals has succeeded in getting its second RNAi drug — for treatment of rare, inherited liver diseases — approved by the European Medicines Authority just months after achieving US FDA approval for the same drug.
Alnylam are well known for being pioneers in the RNA interference space. The company achieved the first ever RNAi drug approval in 2018 with Onpattro (patisiran), in both the US and Europe, after 20 years of research and several setbacks along the way.
Givlaari (givosiran) was approved by the EMA to treat the family of rare inherited liver diseases known as acute hepatic porphyrias in adults and children older than 12 years. Patients with one of these conditions have sudden attacks that can involve stomach pain, vomiting and seizures. These are due to the unnatural build up of a substance called porphyrin, which helps form the iron carrying complex ‘heme’ in the blood, around the body.
Patients normally have a genetic mutation in one of the genes that make heme – a key component of hemoglobin in the blood. This prevents them from producing certain enzymes that allow porphyrin to be processed properly and incorporated into the blood so it builds up in different places around the body instead.
Givlaari acts by reducing the production of a protein associated with porphyrin and heme called aminolevulinic acid synthase 1, which is seen in higher than normal levels in these patients.
The EMA approval stems from a successful phase III trial carried out by Alnylam in 94 patients with acute hepatic porphyria that showed a 74% reduction in the rate of attacks in patients given the drug versus placebo. Patients also reported a reduction in pain symptoms when taking Givlaari.
“Today’s approval of Givlaari marks a historic moment for patients and families living with this devastating genetic disease, as there are currently no approved medicines in Europe proven to decrease the frequency of attacks and reduce the chronic pain that many patients suffer,” said John Maraganore, CEO of Alnylam.
Givlaari, which is given by injection, was given Priority Medicines and Orphan Designation by the EMA and an accelerated review, which is reserved for drugs to treat conditions with serious unmet medical needs. It was reviewed in a similar fashion by the FDA and approved in the US in November 2019.
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