Rare Disease & Special Access

Orphan Care

Rare-disease and orphan-medicine requests often require a named-patient or special-access pathway. Eviga coordinates product identification, supplier checks and document review, while the treating clinician and destination authority remain responsible for medical and regulatory approval.

What we review before supply

• Requests are assessed case by case and are not automatically orderable.
• Clinician justification or authority permission may be required.
• Lead time depends on verified supply and destination documentation.

Orphan Care catalogue

• Pirfenex 200mg (Pirfenidone)

  Antifibrotic agent indicated for idiopathic pulmonary fibrosis under designated orphan care.

• Risdiplam (Generic Reference: Evrysdi)

  Oral solution for Spinal Muscular Atrophy. Procurement via Named Patient Programme.

• Elexacaftor/Tezacaftor/Ivacaftor (Generic Reference: Trikafta)

  Triple combination therapy for Cystic Fibrosis. Highly specialized procurement route.

• Eltrombopag 50mg (Generic Reference: Revolade)

  TPO receptor agonist for chronic immune thrombocytopenia (ITP).

• Lonafarnib (Generic Reference: Zokinvy)

  Treatment for Hutchinson-Gilford Progeria Syndrome.

• Onasemnogene abeparvovec (Generic Reference: Zolgensma)

  Adeno-associated virus vector-based gene therapy for SMA.

• Iptacopan (Generic Reference: Fabhalta)

  First oral monotherapy for adults with PNH.

• Trientine Hydrochloride (Generic Reference: Syprine)

  Copper-chelating agent for Wilson disease.

• Emicizumab (Generic Reference: Hemlibra)

  Bispecific factor for Hemophilia A.

• Imiglucerase (Generic Reference: Cerezyme)

  Enzyme replacement therapy for Type 1 Gaucher disease.

• Eculizumab (Generic Reference: Soliris)

  Complement inhibitor for PNH.

• Nusinersen (Generic Reference: Spinraza)

  SMN2-directed antisense oligonucleotide.

• Vosoritide (Generic Reference: Voxzogo)

  Treatment for achondroplasia.

• Omaveloxolone (Generic Reference: Skyclarys)

  First approved treatment for Friedreich ataxia.

• Tofersen (Generic Reference: Qalsody)

  Treatment for SOD1-ALS.

• Trofinetide (Generic Reference: Daybue)

  Treatment for Rett syndrome.

• Vutrisiran (Generic Reference: Amvuttra)

  RNAi therapeutic for hATTR amyloidosis.

Product information is for catalogue and sourcing purposes and is not medical advice. A prescription and destination-specific approval may be required.