Duchenne muscular dystrophy treatment receives Rare Pediatric Disease designation

The FDA has granted a Rare Pediatric Disease Designation to Avidity Biosciences' AOC 1044, an investigational therapy to treat Duchenne muscular dystrophy (DMD) in patients with mutations amenable to exon 44 skipping (DMD44), according to a press release from Avidity.¹

Typically starting at a young age, DMD is a rare genetic condition that is characterized by progressive muscle damage and weakness because of the loss of dystrophin protein. There are no current approved therapies that target exon 44, according to Avidity Biosciences.¹

Fast Track Designation and Orphan Designation was previously granted to AOC 1044.¹

"We are pleased that the FDA has granted Rare Pediatric Disease designation to AOC 1044, adding to the Orphan Drug and Fast Track designations already granted," said Steve Hughes, MD, chief medical officer, Avidity, in the press release.¹

"The effects of DMD44 are devastating, with symptoms often starting in childhood. These designations by the FDA underscore the urgent need for innovative treatments and validate the potential of AOC 1044 to address the unmet need of people living with DMD," added Hughes.

"We recently shared healthy volunteer data of AOC 1044 from our Phase 1/2 EXPLORE44 trial demonstrating unprecedented delivery of therapeutic oligonucleotide in skeletal muscle and consistent exon skipping in healthy volunteers, and we look forward to sharing data from that study in people living with DMD44 later this year."¹

Positive data for AOC 1044 in healthy individuals was reported in December 2023 from the ongoing EXPLORE44 phase 1/2 trial (NCT05670730).^1,2

The trial is expected to enroll approximately 40 healthy volunteers and 24 patients living with DMD44 aged 7 to 27 years, according to the press release.¹

The treatment is designed to deliver "phosphorodiamidate morpholino oligomers (PMO) to skeletal muscle and heart tissue to specifically skip exon 44 of the dystrophin gene to enable dystrophin production."¹

In the healthy individuals, delivered concentrations of PMO in skeletal muscle with up to 50-times greater concentrations of PMO in skeletal muscle were observed after a single dose compared to peptide conjugated PMOs in healthy individuals.¹

Compared to placebo, AOC 1044 demonstrated statistically significant exon 44 skipping of up to 1.5% in healthy volunteers after a single dose of 10 mg / kg and increased exon 44 skipping in all participants. Overall AOC 1044 was well tolerated.¹

The company expects to provide new AOC 1044 data from people living with DMD44 in the second half of 2024.¹

Related content:

FDA approves vamorolone to treat Duchenne muscular dystrophy

Vamorolone, oral suspension 40 mg / ml, has been approved by the FDA to treat Duchenne muscular dystrophy (DMD) in patients aged 2 years and up, according to Santhera Pharmaceuticals.

Click here to read the full article.

References:

1. Avidity Biosciences receives FDA Rare Pediatric Disease Designation for AOC 1044 for treatment of Duchenne muscular dystrophy in people with mutations amenable to exon 44 skipping. AVidity Biosciences, Inc. Press release. February 20, 2024. Accessed February 22, 2024. https://aviditybiosciences.investorroom.com/2024-02-20-Avidity-Biosciences-Receives-FDA-Rare-Pediatric-Disease-Designation-for-AOC-1044-for-Treatment-of-Duchenne-Muscular-Dystrophy-in-People-with-Mutations-Amenable-to-Exon-44-Skipping

2. Study of AOC 1044 in healthy adult volunteers and participants with Duchenne muscular dystrophy (DMD) mutations amenable to exon 44 skipping (EXPLORE44). ClinicalTrials.gov. Updated February 9, 2024. Accessed February 22, 2024. https://www.clinicaltrials.gov/study/NCT05670730?term=NCT05670730&rank=1