NHS cleared to use AZ's Fasenra for rare autoimmune disease

AstraZeneca's Fasenra has been cleared for use by the NHS in England as an add-on treatment for the rare autoimmune disease eosinophilic granulomatosis with polyangiitis (EGPA).

The final draft guidance (PDF) from reimbursement authority NICE gives a green light to the use of the IL-5 inhibitor in adults with relapsed or refractory EGPA, making it the first biologic therapy for the disease.

EGPA causes vasculitis – inflammation in the wall of blood vessels – and is characterised by asthma, high levels of eosinophils, and inflammation of small- to medium-sized blood vessels. It was previously known as Churg-Strauss syndrome.

First-line treatment is usually with oral corticosteroids, with immunosuppressant drugs in more severe cases, and Fasenra (benralizumab) can now be added to treatment for relapsed/refractory patients. However, use of the biologic needs to stop after 52 weeks if patients don't respond to the combination.

The only other approved biologic for EGPA in the UK is GSK's IL-5 inhibitor Nucala (mepolizumab), which was cleared for marketing several years ago, but was not put forward for appraisal by NICE as a treatment for the disease.

NICE's Fasenra decision is based on the MANDARA trial, which compared the efficacy and safety of AZ's drug with Nucala and showed that it was at least as effective at achieving remission, with that objective reached in around 60% of patients. In addition, treatment with Fasenra allowed 40% of patients to end treatment with steroids, which can have serious side effects if used long-term.

Claire Tolliday, chair of patient group Vasculitis UK, welcomed the decision, noting that living with EGPA has "a profound impact on quality of life, leaving many struggling with even the most basic daily tasks." She added: "EGPA often goes undiagnosed for years, and treatment options have been limited. Today's decision marks an important milestone for patients."

Bad news for Batten disease patients

There was disappointing news today for patients with another rare condition, a form of Batten disease, after NICE determined that it was unable to recommend treatment with BioMarin's enzyme replacement therapy Brineura (cerliponase alfa).

Final draft guidance has concluded that Brineura – which has a list price of around £500,000 per patient per year – is not cost-effective for routine use by the NHS as a treatment for neuronal ceroid lipofuscinosis type 2 (CLN2), a rare and life-limiting form of the disease.

That restriction applies to newly diagnosed patients, however, and patients who have already started treatment will continue to be eligible for the drug – at least until a current commercial agreement between BioMarin and NHS England expires in December.

"Today's final draft guidance comes after the company's decision not to enter into commercial negotiations with NHS England following our appraisal committee meeting last month," said NICE.

Around 30 to 50 children in the UK are living with CLN2, and there are generally between three and six new diagnoses annually.