China’s First In-Hospital Prescription of Oral Factor B Inhibitor Iptacopan Hydrochloride Issued at GoBroad for PNH

On August 31, 2026, the Anemia Diagnosis and Treatment Center at Beijing GoBroad Boren Hospital, part of GoBroad Healthcare Group (GoBroad), issued China’s first in-hospital prescription of iptacopan hydrochloride tablets (brand name: Yishining®), a domestically developed oral Factor B inhibitor, for an adult patient with paroxysmal nocturnal hemoglobinuria (PNH) who had not previously received complement inhibitor therapy.

Iptacopan hydrochloride was approved in China in June 2026. The first in-hospital prescription at Beijing GoBroad Boren Hospital marks the introduction of this new complement-targeted treatment option into routine hospital-based clinical practice for eligible patients with PNH.

First in-hospital prescription: improvement in hematologic parameters after treatment

The first patient was a woman in her 50s. In April 2026, following an episode of gastroenteritis, she noticed dark-colored urine and developed fatigue. Tests at a local hospital showed a hemoglobin level in the 80 g/L range together with an elevated reticulocyte percentage. Further investigations confirmed a diagnosis of PNH.

After diagnosis, the patient reviewed available treatment options, including intravenous C5 inhibitors and oral complement-pathway inhibitors. Following the approval of iptacopan hydrochloride, she visited the Anemia Diagnosis and Treatment Center at Beijing GoBroad Boren Hospital.

After a comprehensive clinical assessment, Dr. Liping Jing and her team determined that the patient met the approved treatment criteria. An outpatient prescription was issued in early July, and on August 31, the team issued China’s first in-hospital prescription.

After more than one month of treatment, the patient’s hemoglobin increased from the 80 g/L range to 144 g/L. Lactate dehydrogenase (LDH) decreased from nearly 2,000 U/L to within the normal reference range, while the reticulocyte count also returned to the normal range. Taken together, these laboratory findings indicated improved control of hemolysis and improvement in anemia compared with baseline.

On the same day, the team issued a second prescription of iptacopan hydrochloride for another patient with PNH. This patient had been diagnosed with aplastic anemia five years earlier and later developed classic PNH. Despite previous treatment, hemoglobin had remained at approximately 100 g/L. After a specialist reassessment and more than one month of treatment with iptacopan hydrochloride, the hemoglobin level increased to 150 g/L, with improvement in anemia-related parameters.

These cases reflect the clinical outcomes of individual patients under specialist evaluation and treatment. They should not be interpreted as predicting the same response in all patients.

From intravenous therapy to oral treatment: expanding options for PNH

PNH is a rare acquired clonal hematopoietic stem cell disorder and is included in China’s first national list of rare diseases. Loss of glycosylphosphatidylinositol (GPI)-anchored proteins on affected blood cells makes them vulnerable to complement-mediated destruction, which may lead to intravascular hemolysis, anemia, hemoglobinuria and thrombosis.

With the development of complement-targeted therapies, the management of PNH has become increasingly individualized.

C5 inhibitors act downstream in the complement cascade and primarily suppress complement-mediated intravascular hemolysis. Iptacopan hydrochloride targets Factor B in the alternative complement pathway, acting at an upstream point in the pathway and providing another mechanism-based treatment option for PNH.

The drug is administered orally twice daily, offering a different mode of administration from intravenous complement inhibitors and expanding treatment choices for patients requiring long-term disease management.

Dr. Jing noted that disease manifestations, prior treatment, complication risks and long-term management needs vary considerably among patients with PNH. The availability of therapies with different mechanisms and routes of administration allows physicians to develop more individualized treatment strategies following a comprehensive assessment.

Accelerating access to new therapies for rare blood disorders

Approval is only the first step in bringing an innovative therapy into clinical practice. Timely hospital evaluation, formulary access, pharmacy management and structured follow-up are equally important in ensuring appropriate use.

As a hospital within GoBroad Healthcare Group with a strong focus on hematologic diseases, Beijing GoBroad Boren Hospital has continued to strengthen its processes for the evaluation, introduction, clinical use and long-term monitoring of innovative therapies for rare blood disorders.

The first in-hospital prescription of iptacopan hydrochloride was issued just over two months after the drug’s approval in China. Supported by GoBroad’s hematology expertise, pharmacy management system and research-oriented medical platform, its hospitals continue to facilitate the structured introduction of new treatment options into clinical practice.

Dr. Jing said: “For patients with a rare disease such as PNH, having more treatment options allows physicians to better tailor therapy to each patient’s disease characteristics, prior treatment and long-term management needs. Once a new therapy enters clinical practice, appropriate use, response monitoring and long-term follow-up remain essential.”

From regulatory approval to the first in-hospital prescription in China, new treatment options are entering PNH clinical practice at an increasingly rapid pace. GoBroad will continue to follow advances in rare hematologic diseases and support the appropriate integration of clinically meaningful innovations into real-world care, with the aim of expanding individualized treatment options for patients.