Alnylam Pharmaceuticals’ new treatment for a rare disease has been shown to be effective — but analysts are now worried about its safety.
The company recently reported that its trial of givosiran, a breakthrough medication that uses RNA interface to target and “silence” specific genes, had shown that the treatment was successful in lowering attacks in patients with acute intermittent porphyria (AIP). The rare disease impacts the liver and can cause severe abdominal pain and neurological symptoms.
But for a large number of patients, givosiran also triggered serious renal impairment and elevated liver enzymes.
On a recent conference call, the company attributed the negative side effects to underlying health conditions. Yet, analysts expressed concerns that the company had not provided sufficient data on the side effects.
Alnylam is moving ahead with its regulatory submissions and hopes to launch the drug in 2020.
Read the full Reuters report.