FDA Approves Givosiran for Adult Patients with Acute Hepatic Porphyria

Katie Ramirez
November 22, 2019

A person born with this genetic disorder can have severe complications ranging from getting acute attacks, commonly referred to as porphyria attacks that may further cause respiratory failure, extreme pain, seizures, paralysis and even lead to changes in the mental health and mental status. Givlaari's annual wholesale price tag will be $575,000, according to Bloomberg. "This prevalence-based adjustment framework may become a model approach for ultra-rare diseases where few or no therapies have previously existed and where diagnosis rates are uncertain".

Alnylam's other treatment, Onpattro, previous year became the first approved treatment that uses gene silencing technology. 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. Assuming the current estimates are right, the company can expect to receive the price that has been demanded. "If, on the other hand, as we improve education, improve disease awareness, if the prevalence goes up a lot, we're willing to take the price down, effectively".

Setting up these types of agreements is "not trivial", Maraganore said. "We believe the approval of Givlaari represents a landmark event for the advancement of precision genetic medicines, providing new hope for patients and their caregivers living with the debilitating manifestations of AHP and unpredictable nature of AHP attacks, as well as for the doctors who diagnose and treat these patients", John Maraganore, PhD, chief executive officer of Alnylam Pharmaceuticals, said in a statement. FDA's approval of givosiran was based on the results of a clinical trial involving 94 patients with acute hepatic porphyria. There was about a 74% reduction in the attacks over the time period of six months on an average. The results in both of the groups were observed by looking at the frequency of porphyria attacks in each of the patients.

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The US Food and Drug Administration (FDA) has approved givosiran (Givlaari) for the treatment of adult patients with acute hepatic porphyria.

Despite the relatively small number of eligible patients, some analysts were projecting peak sales of up to $600 million before Wednesday's approval; Alnylam's own presentation suggested peak revenue for the drug could reach $500 million. The drug is the first to use a type of sugar called GalNAc that allows more precise delivery of drugs to liver cells - which could, potentially, reduce serious side effects associated with the drug going elsewhere in the body.

"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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