Sanofi Genzyme and Alnylam Pharmaceuticals have submitted a marketing application to the European Medicines Agency for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of adults with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis).
The drug has been granted accelerated assessment by the EMA, which could reduce evaluation time from 210 to 150 days.
hATTR amyloidosis is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene, which cause abnormal amyloid proteins to accumulate and damage body organs and tissue, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy.
According to Sanofi and Alynylam, the condition represents “a major unmet medical need with significant morbidity and mortality,” affecting around 50,000 people worldwide who have a life expectancy of 2.5 to 15 years from symptom onset. Currently, the only approved treatment options are liver transplantation for early stage disease and tafamidis.
The submission contains data from the Phase III APOLLO trial, enrolled 225 hATTR amyloidosis patients with polyneuropathy, representing 39 genotypes, at 44 study sites in 19 countries around the world.
At 18 months, the mean change from baseline in the modified neuropathy impairment score (mNIS+7) was significantly lower in the patisiran group as compared with placebo, thus meeting the study’s primary goal.
Also, patients in the patisiran group experienced improvement in quality of life compared to placebo, as assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN), while all five other secondary endpoints also demonstrated statistically significant favourable differences in the treatment arm, including a 10-meter walk test, assessing gait speed and muscle strength.
"Based on the results of the APOLLO study, we believe patisiran has the potential to become the standard of care for the treatment of hATTR amyloidosis.,” said Eric Green, vice president and general manager of the TTR program at Alnylam. “We look forward to working with the EMA and the Committee for Medicinal Products for Human Use (CHMP) during the review process."
"People with hATTR amyloidosis have limited treatment options," added Rand Sutherland, therapeutic area head, Rare Diseases Development at Sanofi. "With this MAA submission, we are one step closer to making patisiran available in Europe and executing on our shared vision to bring this RNAi treatment to patients globally."
Pending regulatory approvals, Alnylam will commercialise the drug in the US, Canada and Western Europe, with Sanofi Genzyme commercialising the product in the rest of the world.