FDA Approves First Acute Hepatic Porphyria Drug

22 Novembre, 2019, 07:37 | Auteur: Therese Cote

Each vial of the drug will cost $39,000.

The RNA interference (RNAi), allows the scientists to mute the genes that do not function appropriately.

Alnylam is facing a major challenger to its first approved drug, Onpattro, from Pfizer's Vyndaqel just over a year into its launch.

The same will be true for givosiran, Alnylam CEO John Maraganore told STAT - but this drug will also be subject to a prevalence-based adjustment. That means that if a given plan winds up covering a higher-than-expected proportion of people with the condition, Alnylam will pay a rebate to the insurer.

"That's never been done before in the rare disease space, but it's something that we think is a responsible and appropriate way to think about ultra-rare, orphan drug pricing", Maraganore said.

"We don't really know what the prevalence is of the disease". He further added that if the company takes an effort to raise awareness regarding the disease and improve education and an increase in the prevalence, there could be an effective reduction in prices. For one thing, it makes it more hard to estimate the amount of money Alnylam will bring in. Tied to the approval of Givlaari, Alnylam announced a new framework for value-based agreements to help patients gain access to the treatment.

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Under VBAs, participating government and commercial payers will pay the full value for GIVLAARI only when it delivers patient outcomes in the real-world setting similar to results demonstrated in clinical trials. There was about a 74% reduction in the attacks over the time period of six months on an average.

Adverse events on the whole occurred at roughly the same rate in both groups, in 90% of Givlaari patients and 80% of placebo patients.

Currently, Anderson said, the only treatment that can sometimes be used is weekly doses of hemin; hemin can't be used to prevent attacks. But the company now has another drug approval to bolster its portfolio--although analysts have expressed some concern about its safety profile.

Recently, the U.S. Food Drug and Administration has given approval for the use of a drug known as Givlaari (givosiran) in treating a rare genetic disease called Acute Hepatic Porphyria in which a patient has a buildup of toxic porphyrin molecules in the blood during the production of heme that is needed for successfully binding the oxygen molecules in red blood cells.

"While these patients have baseline liver disease, and can be attributable to some of the underlying safety events, the more than double SAEs exhibited in the treatment arm compared to placebo could be signs of a safety signal", the analysts wrote.

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