Fabhalta (iptacopan) Now Available from Onco360 as the First FDA-Approved Oral Monotherapy Approved for the Treatment of Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Media Hub / December 20, 2023

Onco360®, the nation’s leading independent Specialty Pharmacy, has been selected as a pharmacy partner by Novartis for Fabhalta® (iptacopan) as the first oral monotherapy approved for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).1 Fabhalta® is a Factor B inhibitor that acts proximally in the alternative complement pathway of the immune system, providing control of red blood cell destruction within and outside the blood vessels (intra- and extravascular hemolysis).

“Onco360 is grateful for the opportunity to partner with the team at Novartis and become a specialty pharmacy provider for Fabhalta®,” said Benito Fernandez, Chief Commercial Officer. “We are proud to add the first oral treatment option for PNH patients.”

Patients with PNH have an acquired mutation that make RBC susceptible to premature destruction by the complement system.2 PNH is characterized by bone marrow failure, hemolysis, and thrombosis in varying levels of severity and combinations. Every year, roughly 500 patients in the U.S. are diagnosed with PNH.3 An estimated 10-16 cases per million patients around the world have PNH. Most patients are diagnosed with PNH in their 30s and 40s.

For more information, please refer to the press release from Novartis here.

Please see the full Prescribing Information for Fabhalta, including Boxed WARNING and Medication Guide.®

About Onco360 Oncology Pharmacy:

Onco360 is the nation’s largest independent Oncology Pharmacy and clinical support services company. Onco360 was founded in 2003 to bring together the stakeholders involved in the cancer treatment process and serve the specialized needs of oncologists, patients, hospitals, cancer centers of excellence, manufacturers, health plans, and payers. It dispenses nationally through its network of URAC-, and ACHC-accredited Oncology Pharmacies. Onco360 is headquartered in Louisville, Kentucky, and is a flagship specialty pharmacy brand of PharMerica Corporation, a leading institutional pharmacy, specialty infusion, and hospital services company servicing healthcare facilities in the United States. For more information about Onco360, please visit Onco360.com.


  1. Iptacopan Prescribing Information. East Hanover, NJ: Novartis Pharmaceuticals Corp; December 2023.
  2. Cançado RD, Araújo A da S, Sandes AF, et al. Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria. Hematol Transfus Cell Ther. 2021;43(3):341-348. doi:10.1016/j.htct.2020.06.006
  3. Shah N, Bhatt H. Paroxysmal Nocturnal Hemoglobinuria. [Updated 2023 Jul 31]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2023 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK562292/
  4. Risitano AM, Han B, Ueda Y, et al. Oral Complement Factor B Inhibitor Iptacopan Monotherapy Improves Hemoglobin to Normal/Near-Normal Levels in Paroxysmal Nocturnal Hemoglobinuria Patients Naïve to Complement Inhibitors: Phase III APPOINT-PNH Trial. Presented at: 49th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT); April 23-36, 2023; Paris, France.
  5. Risitano AM, Rӧth A, Kulasekararaj A, et al. Oral Iptacopan Monotherapy Has Superior Efficacy to Anti-C5 Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria and Residual Anemia: Results from the Phase III APPLY-PNH Study. Presented at: 49th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT); April 23-36, 2023; Paris, France.

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