Oral Orladeyo Now Available in UK to Treat HAE Swelling Attacks

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by Marisa Wexler MS |

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Orladeyo (berotralstat), a daily oral therapy to prevent swelling attacks in people with hereditary angioedema (HAE), is now available in England, Wales, and Northern Ireland for eligible patients ages 12 and older.

That authorization came as the U.K.’s National Institute for Health and Care Excellence (NICE) recommended Orladeyo — marketed by BioCryst Pharmaceuticals — as a treatment for people with HAE who have at least two swelling attacks per month.

The Scottish Medicines Consortium (SMC) is expected to issue a decision on Orladeyo’s use for patients in Scotland under the U.K.’s National Health Service (NHS) in the first half of next year.

“We are excited for HAE patients that this recommendation from NICE provides access to the first oral, once-daily treatment for UK patients to achieve symptom control and experience relief from the burdens of HAE,” Charlie Gayer, chief commercial officer of BioCryst, said in a press release.

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Gayer added that NICE’s decision “expands access to modern prophylaxis [preventive treatment] with Orladeyo, compared to the attack frequency requirements from NICE for injectable options.”

Orladeyo was approved by the European Commission in April, and the U.K.’s Medicines and Healthcare products Regulatory Agency issued a similar approval the following month.

It also has been approved in the U.S. and Japan, and is being reviewed by regulatory authorities in Canada, Switzerland, and Israel.

These approvals, and NICE’s recommendation, were supported by data from the Phase 3 APeX-2 trial (NCT03485911), sponsored by BioCryst, in which 121 people with HAE were given Orladeyo or a placebo for about six months.

Results showed that treatment with the medication sustainably reduced the mean rate of monthly swelling attacks by approximately 80% over the course of nearly two years (96 weeks).

Orladeyo also is being evaluated in an open-label Phase 2/3 trial, called APeX-S (NCT03472040). Interim data from this trial have supported the medication’s favorable safety profile, and also showcased its ability to lower the rate of swelling attacks among patients.

Laura Szutowicz, CEO of the charity organization HAE U.K., said her advocacy group “welcomes the NICE decision on berotralstat, which means that eligible patients and clinicians have another choice of treatment for this lifelong condition.”

“The NICE recommendation of berotralstat is great news for clinicians and eligible patients who now have access to the first oral preventive treatment for HAE that could significantly reduce the number of attacks and may improve quality of life,” said Sorena Kiani, PhD, a consultant immunologist at Barts Health NHS Trust.