Latest news

• Summit plunges after Duchenne disappointment

  These include the use of CRISPR/Cas9, myostatin Inhibitior candidates from Pfizer and BMS, and Santhera which is developing  Idebenone (Raxone).

• CHMP hands Santhera second ‘no’ in Duchenne muscular dystrophy

  European regulatory advisers have refused to shift their negative position on Santhera’s Duchenne muscular dystrophy (DMD) drug Raxone (idebenone).

• Santhera facing long delay for muscular dystrophy drug in US

  The Swiss biopharma company has failed to convince the FDA to accept an accelerated marketing application for Raxone (idebenone) via the truncated subpart H approval pathway, used to hasten the approval

• BioMarin gives up on muscular dystrophy drug

  There has been some good news for European DMD patients this week however after Santhera announced it had filed for approval of Raxone (idebenone) for the treatment of DMD.

• Ten new medicines recommended in Europe

  And finally the CHMP has recommended Santhera's Raxone (idebenone) for the treatment of visual impairment in patients with Leber's hereditary optic neuropathy. .