Agency backs NHS coverage of Rystiggo for hard-to-treat gMG

The National Institute for Health and Care Excellence (NICE) has recommended that Rystiggo (rozanolixizumab-noli) be covered by England’s National Health Service (NHS) and provided at low or no cost to certain adults with hard-to-treat generalized myasthenia gravis (gMG).

The recommendation makes Rystiggo the first gMG therapy to be recommended by NICE. A draft version of the new recommendation was made public in late April, with final publication expected in the coming weeks.

“Generalized myasthenia gravis can severely limit people’s independence and place a heavy burden on patients, families and carers,” Helen Knight, director of medicines evaluation at NICE, said in an agency press release. “Today’s draft recommendation marks an important step forward for people living with the condition when it remains uncontrolled on current treatment.”

NICE said Rystiggo coverage will be immediately available to eligible patients: those testing positive for the most common gMG-driving self-reactive antibodies and whose disease remains uncontrolled after trying at least two standard medications.

This is a narrower indication than the approved indication in the U.K., where Rystiggo is cleared for use as an add-on to standard treatment for adult gMG patients who are positive for the most common antibodies.

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gMG is an autoimmune disease marked by self-targeting antibodies that interfere with signaling from nerve to muscle cells, resulting in symptoms such as fatigue and muscle weakness in several parts of the body. The most common gMG-driving antibodies target one of two proteins, acetylcholine receptor (AChR) or muscle-specific tyrosine kinase (MuSK).

Rystiggo, sold by UCB, is approved in the U.K., as well as the U.S. and European Union, to treat adults with gMG who are positive for antibodies against AChR and/or MuSK. The therapy is given via an under-the-skin infusion pump, once per week in six-week cycles, with additional cycles given as needed to manage symptoms.

Rystiggo works to lower gMG-driving antibody levels by blocking the activity of a protein that normally helps prevent circulating antibodies from being degraded. The therapy’s approvals were based mainly on data from MycarinG (NCT03971422), a Phase 3 clinical trial that demonstrated Rystiggo was significantly better than a placebo at easing symptoms in gMG patients positive for anti-AChR or anti-MuSK antibodies.

Rystiggo “offers a new targeted treatment option with the potential to improve symptoms and help people carry out their day‑to‑day activities,” Knight said.

Per the NICE recommendation, the NHS will cover Rystiggo for adults with gMG and antibodies against AChR or MuSK, and whose disease remains uncontrolled after trying at least two other medications, excluding acetylcholinesterase inhibitors such as pyridostigmine (sold as Mestinon and with generics available). Previously tried medications can include corticosteroids, a type of anti-inflammatory and immunosuppressive medication commonly used in gMG.

Eligible patients are also receiving intravenous immunoglobulin or plasma exchange, two therapeutic approaches designed to neutralize or remove disease-driving antibodies in the blood that generally require frequent trips to a hospital for treatment. If not, they must have tried these treatments but stopped due to side effects or lack of efficacy.

The list price of Rystiggo is £17,883.19 per vial (about $24,201.79). UCB is providing the therapy to the NHS at a discounted price, but the specifics are being kept confidential.

The NICE recommendation notes that there is uncertainty about exactly how cost-effective Rystiggo is, but based on available models, “the cost-effectiveness estimates were likely to be within the range that NICE considers a cost-effective use of NHS resources.”