Alnylam's Givlaari wins NICE reversal after offering discount for rare genetic liver condition therapy

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Alnylam's Givlaari was approved for acute hepatic porphyria two years ago and is provided to those who are stricken with the most severe form in which patients suffer four or more seizures a year. (Alnylam)

Since Alnylam’s Givlaari was approved in Europe in March 2020, those in the U.K. with the rare disorder called acute hepatic porphyria have been waiting to be able to use it.

Nineteen months later, the wait is almost over.

With Thursday’s nod from England’s drug-price watchdog, Givlaari will soon be provided through the National Health Service. The recommendation reverses (PDF) the National Institute for Health and Care Excellence's (NICE's) previous decision to reject the treatment, which was originally priced at $575,000 annually, or $39,000 per vial.

Today's NICE recommendation comes after an Alnylam offer to provide the therapy at a confidential discount.

AHP is a life-threatening genetic condition that strikes the liver with a buildup of pigments called porphyrins that can lead to nausea, vomiting and seizures. It can lead to a host of other disorders including paralysis, insomnia and heart and lung problems.

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England’s National Acute Porphyria Service (NAPS) hailed today's decision. “We’re delighted that NICE has issued draft guidance recommending the use of (Givlaari) on the NHS,” the organization said on Twitter. “This is a huge development for patients.”

Givlaari is provided to those who are stricken with the most severe form of AHP in which patients suffer four or more seizures a year. Another drug prescribed for the condition—Normosang from Italian pharmaceutical company Recordati—loses its effectiveness with a few years of use, NICE said.

AHP hits 1 in 100,000 people in Europe or 560 in England. Most recover after one attack but recurrent attacks occur in roughly 10% of patients. According to NAPS, 27 people in the U.K. have recurring attacks and 17 new cases are diagnosed annually.

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“It is hard to overstate the life-limiting impact of this condition on patients and their families. Some patients experience regular, intense pain, have to spend long periods of time in hospital and are unable to continue working or have a normal social life," David Rees, professor haematology at King's College London, said in a statement. "By using this pioneering ‘gene silencing’ approach, we can now target the production of toxic compounds that have the potential to wreak havoc in the body, tackling the cause of a patient’s attacks at the source.”

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