28 FEBRUARY 2025

The Medicines and Healthcare products Regulatory Agency (MHRA) has approved Italfarmaco’s Duvyzat (givinostat) to treat Duchenne muscular dystrophy (DMD) in patients aged six years and older.

The regulator has specifically granted full approval of the drug for patients who are beginning treatment when ambulatory, and has issued a conditional approval for those who start treatment when no longer ambulatory.

Estimated to affect approximately 2,500 people in the UK at any one time, DMD is a rare neuromuscular disease caused by a change or mutation in the gene that encodes instructions for dystrophin, which is required to strengthen and protect muscles.

Duvyzat is an orally administered histone deacetylase inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle.

The MHRA’s decision references the US Food and Drug Administration’s approval of the drug in March 2024 and was granted through the International Recognition Procedure.

Supporting both applications were positive results from the late-stage EPIDYS trial of 179 ambulant boys, with Duvyzat-treated patients demonstrating a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment.

Biogen has announced that the European Medicines Agency (EMA) and US Food and Drug Administration (FDA) have started reviewing a higher dose regimen of its spinal muscular atrophy (SMA) treatment Spinraza (nusinersen).

The antisense oligonucleotide is already approved in more than 71 countries at a dose of 12mg to treat infants, children and adults with the neuromuscular disease.

The company is now seeking approval for a “more rapid” loading regimen of two 50mg doses two weeks apart, compared to the current four loading doses, and a higher maintenance regimen of 28mg every four months.

Affecting approximately one in every 10,000 babies worldwide, SMA is a rare, progressive genetic disorder characterised by insufficient levels of the survival motor neuron (SMN) protein. Symptoms vary, but can include muscle weakness, problems with breathing and swallowing, and tremors.

Biogen’s Spinraza targets the underlying cause of motor neuron loss by continuously increasing the amount of full-length SMN protein produced in the body.

The company’s applications for its higher dose regimen were supported by positive results from the phase 2/3 DEVOTE study of 145 SMA patients.

Eli Lilly and Alchemab Therapeutics have partnered to develop new therapies for amyotrophic lateral sclerosis (ALS).

The collaboration agreement centres around Alchemab’s antibody discovery platform, which uses patient samples from those with unusually slow rates of disease progression to identify antibodies associated with resilience.

Alchemab has already acquired hundreds of ALS samples, and both companies will work together to discover, develop and commercialise up to five novel therapeutics using the platform.

In exchange, Alchemab will receive an undisclosed upfront fee and will be eligible to receive discovery, development and commercialisation milestone payments, as well as royalties.

Affecting an estimated 30,000 people in the US, ALS is a rare and fatal neurodegenerative disease for which there is no known cure. The disorder results in the progressive loss of motor neurons that control voluntary muscles, leading to difficulties in speaking, swallowing, walking and breathing.

Alchemab’s chief executive officer, Jane Osbourn, said the company has already shown that its differentiated antibody discovery platform “can lead to insights into how an individual’s immune response can generate potent, selective and unique antibodies with therapeutic potential”.

Novo Nordisk has shared positive results from a late-stage study of its investigational haemophilia A therapy Mim8 in patients aged one to 11 years.

The phase 3 FRONTIER3 trial initially evaluated a once-weekly dose of the candidate to prevent prolonged and spontaneous bleeding, before giving patients the option to move to once-monthly dosing after 26 weeks.

Approximately 1,125,000 people worldwide are affected by haemophilia, a rare inherited bleeding disorder that impairs the body’s ability to make blood clots. Haemophilia A, which is caused by a missing or defective clotting factor VIII, accounts for up to 85% of global cases.

Novo’s Mim8 is a subcutaneously administered mimetic bi-specific antibody that is designed to replace the function of the missing clotting factor VIII.

The interim FRONTIER3 data demonstrated that Mim8 was well tolerated and efficacious in patients with and without inhibitors.

For part one of the study, which assessed once-weekly dosing, the estimated mean annualised bleeding rate (ABR) for treated bleeds was 0.53 and the median ABR was zero, with 74.3% of patients experiencing zero treated bleeds.

The US Food and Drug Administration (FDA) has approved SpringWorks Therapeutics’ Gomekli (mirdametinib) to treat neurofibromatosis type 1 (NF1), a rare genetic disorder affecting approximately 100,000 people in the US.

The drug has been specifically authorised to treat adult and paediatric patients aged two years and older who have symptomatic plexiform neurofibromas (PN) that cannot be removed surgically.

NF1 causes a variety of symptoms across multiple organ systems, including abnormal pigmentation, skeletal deformities, tumour growth and neurological complications.  Around 40,000 NF1 patients in the US have PNs, which are tumours that grow along the peripheral nerve sheath and are associated with severe disfigurement and pain.

SpringWorks’ Gomekli is an oral small molecule MEK inhibitor and the first and only FDA-approved therapy for both adults and children with NF1-PN.

The FDA’s decision on the drug was supported by results from the phased 2b ReNeu trial, which demonstrated an objective response rate of 41% and 52% in adult and paediatric NF1-PN patients, respectively.

Tumour volume reductions were deep and durable, and patients in both cohorts experienced early and sustained improvements in pain and quality of life.