21 December 2019

Zolgensma to be made available globally under a managed-access programme.

Briefly, we understand that:

• In January 2020 they will give their gene therapy drug Zolgensma for free to eligible patients around the world, including Malaysia.
  
• They will give up to 100 doses of Zolgensma per year depending on production capacity, of which 50 doses is expected in the first half of 2020. MAPS is planned to continue after 2020.
  
• Only some patients who apply will be successful to get the free Zolgensma dose, not all.
  
• All children must be below 2 years at the time of dosing.
  

Additional information and guidance were shared.

To be clear, only doctors are permitted to submit requests to Avexis to receive free doses of Zolgensma for their patients.

If you believe your child would be eligible, please contact your doctor.

If you and your doctor need information and guidance on GMAP, please contact TreatSMA@WeCareJourney.org, or Ms Sook Yee on 012-239-5592, or Kak Sha on 012-671-1306. Several representatives from Persatuan WeCareJourney and our Medical Board of Advisors were invited to and attended the GMAP calls and discussions.

Please help to circulate this letter to all the families with SMA in Malaysia that you are in contact with.

Zolgensma akan disediakan di peringkat global di bawah program akses terurus.

Pada 17 dan 18 Disember, AveXis mengadakan panggilan taklimat dan perbincangan mengenai Program Akses Terurus Global ("GMAP") yang baru untuk Zolgensma (AVXS-101).

Secara ringkas, kami faham bahawa:

• Pada bulan Januari 2020 mereka akan memberi ubat terapi gen mereka Zolgensma secara percuma kepada pesakit yang layak di seluruh dunia, termasuk Malaysia.
• Mereka akan memberi sehingga 100 dos Zolgensma setahun bergantung kepada kapasiti pengeluaran, di mana 50 dos dijangkakan pada separuh pertama 2020. MAPS dirancang untuk diteruskan selepas 2020.
• Hanya beberapa pesakit yang memohon akan berjaya mendapatkan dos Zolgensma percuma, bukan semua.
• Semua kanak-kanak mesti berada di bawah 2 tahun pada masa dos.

Maklumat dan panduan tambahan dikongsi.