Viltolarsen

MDA Celebrates FDA Approval of Amondys 45 for Treatment of DMD Amenable to Exon 45 Skipping

Retrieved on: 
Thursday, February 25, 2021
Muscular dystrophy, Therapeutic gene modulation, Antisense RNA, Branches of biology, Biotechnology, Biology, Orphan drugs, Eteplirsen, Dystrophin, Viltolarsen, Duchenne muscular dystrophy

Amondys 45 is designed to treat a third subset of patients with DMD, specifically those with a mutation amenable to skipping of exon 45.

Key Points: 
  • Amondys 45 is designed to treat a third subset of patients with DMD, specifically those with a mutation amenable to skipping of exon 45.
  • Approval of Amondys 45 represents another significant step forward in the development of therapies for DMD that target the root cause of the disease.
  • It is estimated that up to 8% of patients with DMD have mutations amenable to treatment with Amondys 45.
  • Just as individuals with DMD caused by a mutation that would be amenable to skipping exon 51 could benefit from treatment with Exondys 51, those with DMD caused by a mutation that would be impacted by skipping exon 45 potentially could benefit from treatment with Amondys 45.

FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation

Retrieved on: 
Thursday, February 25, 2021
Muscular dystrophy, Branches of biology, Duchenne muscular dystrophy, Biology, Viltolarsen, Dystrophin, Exon skipping

This is the first FDA-approved targeted treatment for patients with this type of mutation.

Key Points: 
  • This is the first FDA-approved targeted treatment for patients with this type of mutation.
  • Approximately 8% of patients with DMD have a mutation that is amenable to exon 45 skipping.
  • "Today's approval of Amondys 45 provides a targeted treatment option for Duchenne muscular dystrophy patients with this confirmed mutation."
  • DMD occurs in approximately one out of every 3,600 male infants worldwide; in rare cases, it can affect females.

Option Care Health Selected as a Limited Distribution Provider of VILTEPSO™ (viltolarsen) Injection for Patients with Duchenne Muscular Dystrophy in Patients Amenable to Exon 53 Skipping Therapy

Retrieved on: 
Wednesday, September 2, 2020
Branches of biology, Biology, Viltolarsen, Duchenne muscular dystrophy, Muscular dystrophy, Dystrophin, DMD, Exon skipping, Drisapersen

BANNOCKBURN, Ill., Sept. 02, 2020 (GLOBE NEWSWIRE) -- Option Care Health, Inc. (Option Care Health) (NASDAQ: OPCH), the nations largest independent provider of home and alternate site infusion services, has been selected to participate in the limited distribution network of VILTEPSO (viltolarsen) for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to exon 53 skipping therapy.

Key Points: 
  • BANNOCKBURN, Ill., Sept. 02, 2020 (GLOBE NEWSWIRE) -- Option Care Health, Inc. (Option Care Health) (NASDAQ: OPCH), the nations largest independent provider of home and alternate site infusion services, has been selected to participate in the limited distribution network of VILTEPSO (viltolarsen) for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to exon 53 skipping therapy.
  • Manufactured by NS Pharma, Inc., Viltepso is the first and only exon 53 skipping therapy to demonstrate an increase in dystrophin in patients as young as four years old.
  • It is caused by genetic mutations that prevent the production of dystrophin, a key protein for supporting muscle health.
  • Option Care Health works closely with providers to develop customized patient care plans and provide regular treatment updates.