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Blood disorder medicines from the US: what is approved and how to access it

Hemophilia, sickle cell disease, thalassemia, PNH and more: US-approved options checked against FDA labels on 17 October 2026.

Reviewed by the Reserve Meds clinical operations team on 17 October 2026. Sources accessed 17 October 2026.

The short answer

The US has approved several newer treatments for inherited blood disorders, including preventive injections for hemophilia, one-time gene therapies for hemophilia, sickle cell disease and beta thalassemia, and complement inhibitors for PNH. If one is not registered in your country, the treating hematologist's hospital can request it for the named patient. Gene therapies are given at qualified centers.

  • Conditions covered: Hemophilia A and B, sickle cell disease, beta thalassemia, PNH, PK deficiency, lower-risk MDS anemia.
  • Medicines: Ask the hospital to request an unregistered medicine for the named patient.
  • Gene therapies: Given at qualified centers.
  • Price: Quote on request.

Hemophilia

  • Hemlibra (emicizumab): routine prophylaxis of bleeding episodes in patients from newborn with hemophilia A, with or without factor VIII inhibitors.[1]
  • Qfitlia (fitusiran): routine prophylaxis of bleeding episodes in patients 12 years and older with hemophilia A or B, with or without inhibitors.[2]
  • Alhemo (concizumab): routine prophylaxis of bleeding episodes in patients 12 years and older with hemophilia A or B, with or without inhibitors.[3]
  • Hemgenix (etranacogene dezaparvovec): one-time gene therapy for adults with hemophilia B who use factor IX prophylaxis, or have had life-threatening or repeated serious bleeding.[4]
  • Beqvez (fidanacogene elaparvovec): one-time gene therapy for adults with moderate to severe hemophilia B who meet the label criteria.[5] Pfizer stopped developing and selling Beqvez worldwide in February 2025.
  • Roctavian (valoctocogene roxaparvovec): one-time gene therapy for adults with severe hemophilia A without pre-existing antibodies to AAV5 detected by an FDA-approved test.[6]

Sickle cell disease and beta thalassemia

  • Casgevy (exagamglogene autotemcel): gene-edited cell therapy for sickle cell disease with recurrent vaso-occlusive crises and for transfusion-dependent beta thalassemia.[7]
  • Lyfgenia (lovotibeglogene autotemcel): gene therapy for patients 12 years and older with sickle cell disease and a history of vaso-occlusive events.[8]
  • Zynteglo (betibeglogene autotemcel): gene therapy for adults and children with beta thalassemia who need regular red blood cell transfusions.[9]
  • Reblozyl (luspatercept): anemia in adults with beta thalassemia who need regular transfusions, and anemia in certain adults with lower-risk myelodysplastic syndromes.[10]

In the US, the CMS Cell and Gene Therapy Access Model negotiates outcomes-based terms for sickle cell gene therapies on behalf of Medicaid programs. It does not cover patients outside the US.[11]

PNH, PK deficiency and lower-risk MDS

  • Fabhalta (iptacopan): adults with paroxysmal nocturnal hemoglobinuria (PNH), and certain kidney diseases (IgA nephropathy, C3 glomerulopathy).[12]
  • Empaveli (pegcetacoplan): adults with PNH, and patients 12 years and older with C3 glomerulopathy or primary IC-MPGN.[13]
  • Piasky (crovalimab): patients 13 years and older weighing at least 40 kg with PNH.[14]
  • Pyrukynd (mitapivat): hemolytic anemia in adults with pyruvate kinase deficiency.[15]
  • Rytelo (imetelstat): transfusion-dependent anemia in adults with low- to intermediate-1 risk myelodysplastic syndromes who meet the label criteria.[16]

How patients in the Gulf access these medicines

In Saudi Arabia, the SFDA Special Access Program lets a patient with a serious or immediately life-threatening condition receive a medicine that is not registered when no comparable alternative exists. The request comes from the manufacturer or sponsor, or from the licensed treating physician if there is no sponsor. We give you a written timeline estimate for your case before you commit.[17]

In Bahrain, NHRA Circular 41 of 2020 lets government and private hospitals import a non-registered medicine with the authority's permission before the purchase order, when the medicine is important and has no registered alternative in Bahrain, is registered with the Gulf Health Council, a GCC state or a reference regulator such as the US FDA, and is marketed in its country of origin.[18]

Gene therapies such as Hemgenix, Beqvez, Roctavian, Casgevy, Lyfgenia and Zynteglo are on FDA's list of approved cellular and gene therapy products. They are given once at qualified centers after eligibility testing, which for some includes antibody tests named in the US label.[19, 6]

How Reserve Meds helps

Reserve Meds is a digital health company. Medicines are supplied through a US-licensed pharmaceutical wholesale partner. We help families and treating doctors understand the route, prepare the documents the regulator or hospital asks for, arrange supply from the US once approval is in place, and keep everyone updated. We do not diagnose, prescribe or decide what a regulator approves.

We coordinate access to FDA-approved medicines only. Investigational medicines are only available through a manufacturer's expanded-access program requested by the treating physician; we can explain that route, but we do not supply them. Your coordination fee is quoted in writing for your case before you commit. No hidden charges. We give you a written timeline estimate for your case before you commit.

Ask us about a medicine

Tell us the medicine, your country and a WhatsApp or phone number. We reply within one business day. On WhatsApp we usually reply the same day.

Include the country code, for example +966 or +971.
Message us on WhatsApp

No account needed. We reply within one business day. Your coordination fee is quoted in writing for your case before you commit. No hidden charges.

Frequently asked questions

Is there a one-time gene therapy for hemophilia?

Yes. FDA has approved Hemgenix for hemophilia B and Roctavian for severe hemophilia A, each for patients who meet the criteria in the US label. FDA also approved Beqvez for hemophilia B in 2024, but Pfizer stopped selling it worldwide in February 2025.

Which gene therapies are FDA-approved for sickle cell disease?

Casgevy and Lyfgenia. Casgevy is also approved for transfusion-dependent beta thalassemia, and Zynteglo for beta thalassemia needing regular transfusions.

Can a hemophilia patient in Saudi Arabia get a medicine that is not registered?

Yes, usually through the treating hospital's request to the SFDA Special Access Program.

Are these medicines available at any hospital?

Preventive injections can usually be supplied to the treating hospital once approved. Gene therapies are given only at centers the manufacturer has qualified.

Sources for these answers: [4, 5, 6, 7, 8, 17]

Sources

All sources were opened and checked on 17 October 2026. Rules and labels change, so the treating hospital or regulator confirms the current position before anything is filed.

  1. DailyMed: Hemlibra (emicizumab) US prescribing information, version effective 11 July 2025 (accessed 17 October 2026)
  2. DailyMed: Qfitlia (fitusiran) US prescribing information, version effective 16 September 2025 (accessed 17 October 2026)
  3. DailyMed: Alhemo (concizumab) US prescribing information, version effective 1 July 2025 (accessed 17 October 2026)
  4. US FDA: HEMGENIX product page (CBER) (accessed 17 October 2026)
  5. US FDA: BEQVEZ product page (CBER) (accessed 17 October 2026)
  6. US FDA: ROCTAVIAN product page (CBER) (accessed 17 October 2026)
  7. US FDA: CASGEVY product page (CBER) (accessed 17 October 2026)
  8. US FDA: LYFGENIA product page (CBER) (accessed 17 October 2026)
  9. US FDA: ZYNTEGLO product page (CBER) (accessed 17 October 2026)
  10. DailyMed: Reblozyl (luspatercept) US prescribing information, version effective 27 February 2026 (accessed 17 October 2026)
  11. US Centers for Medicare and Medicaid Services: Cell and Gene Therapy (CGT) Access Model (accessed 17 October 2026)
  12. DailyMed: Fabhalta (iptacopan) US prescribing information, version effective 16 July 2026 (accessed 17 October 2026)
  13. DailyMed: Empaveli (pegcetacoplan) US prescribing information, version effective 21 August 2026 (accessed 17 October 2026)
  14. DailyMed: Piasky (crovalimab) US prescribing information, version effective 11 November 2025 (accessed 17 October 2026)
  15. DailyMed: Pyrukynd (mitapivat) US prescribing information, version effective 30 June 2026 (accessed 17 October 2026)
  16. DailyMed: Rytelo (imetelstat sodium) US prescribing information, version effective 22 April 2026 (accessed 17 October 2026)
  17. SFDA: Special Access Program (SAP) guidance, version 1.0 (accessed 17 October 2026)
  18. NHRA Bahrain: Circular No. (41) of 2020, procedures for importing non-registered medicines (accessed 17 October 2026)
  19. US FDA: Approved Cellular and Gene Therapy Products (accessed 17 October 2026)

Related

This page is general information, not medical or legal advice. It does not replace advice from your own doctor or the regulator.

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