Givlaari Access in England, via NHS, Now Expected by February

Marisa Wexler MS avatar

by Marisa Wexler MS |

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By February, eligible acute porphyria patients in England are expected to have access to Givlaari (givosiran) through the country’s National Health Service (NHS), for treating severe, recurrent disease attacks, according to an update from the British Porphyria Association.

The announcement comes in the wake of a draft guidance issued by England’s National Institute for Care and Excellence (NICE) recommending Givlaari as a treatment for acute porphyria attacks. There now will be a month-long appeal period, followed by three months in which the NHS will take all necessary steps to ensure that patients have access to the treatment.

The medicine is expected to be available to patients by late February, according to the association. It is indicated for individuals who experience severe and recurrent porphyria attacks — meaning those having at least four porphyria attacks over the period of one year.

“For decades, people with this life-threatening condition have had limited options to control the relentless and debilitating pain, nausea, and fatigue that dominate virtually all aspects of their lives. The decision today from NICE is therefore a very welcome and important one,” John Chamberlayne, chair of trustees at the British Porphyria Association, said in a press release.

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Notably, because some people can have allergic reactions to the medicine, Givlaari will initially only be given at expert centers.

Chamberlayne also noted that making Givlaari available “will mark real change for patients and families in future, allowing more people to live without the constant fear of when the next agonising attack may strike.”

Givlaari is an RNA-interference therapy that is designed to reduce the production of the intermediary molecules that build up to toxic levels in porphyria. Clinical trials have demonstrated the treatment is generally safe and can substantially reduce disease burden.

The therapy, marketed by Alnylam Pharmaceuticals, has been approved in the U.S. and Europe as a treatment for acute hepatic porphyria.

“[Porphyria] robbed me of so much: to take part in normal life, the chance to work and develop a career, to walk, run and exercise, to travel,” said Liz, a 44-year-old living with acute intermittent porphyria, the most common form of acute porphyrias.

“Finally, in my mid-40s, [Givlaari] has given me the chance to start experiencing life as it should be,” Liz was quoted as saying.

Givlaari was recently recommended for public reimbursement in Canada when used to treat adults with acute hepatic porphyria under certain conditions.

“There will be strict criteria on adherence to the protocols for treating” patients in England with the medication, the British Porphyria Association said.