Hospital trust to use new “game-changing” drug to treat rare muscle disease

University Hospital Southampton (UHS) is set to become one of the first in the UK to offer a new drug to treat myasthenia gravis (MG) – a rare and serious neuromuscular autoimmune condition.

The drug, efgartigimod alfa, was discovered by Southampton-based researcher Sally Ward, professor of molecular immunology and director of translational immunology at the University of Southampton.

It is considered to be “game-changing” in the treatment of the condition as, up until now, there has been a “high unmet clinical need”.

Myasthenia gravis can develop at any age and causes debilitating and potentially life-threatening muscle weakness.

Young patients with MG are more commonly female, whereas patients over the age of 50 are more often male.

The condition can affect multiple muscle groups throughout the body and causes shortness of breath and extreme fatigue.

It also leads to difficulties in swallowing and with speech and vision, as well as impaired respiratory function.

This is due to high levels of IgG autoantibodies (an immune system protein) in the body that are associated with many autoimmune diseases.

These attack and damage healthy proteins on nerves called acetylcholine receptors which cause errors in the transmission of nerve impulses to the muscles.

Because of this damage the nerves are not able to make the muscles contract as well as normal, leading to muscle weakness and difficulty in moving.

The new drug binds to and blocks the function of a protein in the body called the neonatal Fc receptor (FcRn), leading to a decrease in the level of IgG autoantibodies that are responsible for the damage.

Current standard treatment options include intravenous immunoglobulin (IVIg) – a therapy for patients with antibody deficiencies which is prepared from a pool of immunoglobulins (antibodies) from the plasma of healthy donors – surgery to the thymus gland and steroid treatment.

There are approximately 15 in every 100,000 people living with MG in the UK – and UHS sees around 440 patients every year with the condition.

Mathew Garrod, lead pharmacist for neurosciences at UHS, said: “This new drug is potentially game-changing for patients with myasthenia gravis which can be an extremely debilitating condition.

“Up until now options have been limited. Intravenous immunoglobulin therapy, which relies on plasma donation, is currently a scarce resource under a demand management programme so anything to preserve those stocks is welcome.

“Outcomes of the efgartigimod trials have proven to be very promising. For so long there has been a high unmet clinical need in treatment options that are effective, targeted, well tolerated and can be used in a broad population of adult patients.”

Other benefits include fewer hospital appointments and improved availability of existing IVIg stock for other patients.

Georgina Burke, neurology consultant at UHS, said: “There are many benefits to this new drug, including that it can be administered by a nurse in the patient’s home – avoiding the need for multiple hospital visits.

“It is a cycle of four one-hour infusions every week followed by a treatment break.

“By comparison, IVIg requires a hospital day case appointment of up to five hours for one to five consecutive days followed by a treatment break.

“Results are showing that maximum effect is reached one week after the last infusion of the initial treatment cycle – which is about five weeks after starting treatment.

“The effects appear to last for about seven to nine weeks, but this may vary per patient.”

Patients are able to stay in touch with the clinical team in-between appointments using My Medical Record (MMR) – an online personal health record developed at UHS.

Using the MMR website or app, patients co-manage their care by recording and sending data securely to the clinical team in real-time, meaning clinicians are able to monitor patients’ symptoms and efficacy of the drug.

University Hospital Southampton is currently one of three sites across the UK offering the drug which can be accessed through the Medicines & Healthcare products Regulatory Agency (MHRA) Early Access to Medicines Scheme (EAMS).

The scheme aims to provide earlier availability of promising new medicines to UK patients that have a high unmet clinical need.

Mr Garrod added: “We are extremely pleased that Southampton is among the first sites in the UK to offer this drug to patients through the EAMS and hope that results continue to prove that a new era of drug therapy has now opened up to this group of patients.

“With further research, potentially thousands of other patients suffering with other autoimmune diseases may also benefit from this type of treatment in the future.”

Prof. Ward said: “It is extremely gratifying that our research related to understanding FcRn function and the generation of engineered antibodies that target this receptor has led to an approved drug to treat myasthenia.”

The drug is currently only available for patients over 18 with generalised myasthenia gravis because further clinical trials are required to confirm its safety and efficacy.

It is not available for patients with ocular myasthenia gravis which is a form of the condition that only affects the muscles that move the eyes and eyelids.