CSL Behring

This is a phase 2a, global, multicenter, randomized, double-blind, placebo-controlled study investigating the safety of anumigilimab administered subcutaneously (SC) at the maximum tolerated dose (MTD) in adult participants with SCD. The primary aim of the study is to assess the safety of anumigilimab in participants with SCD. Participants will be treated for 64 weeks: for 12 weeks in the dose escalation period, where the dose will be escalated to each participant's individual MTD; and for 52 weeks at the MTD in the maintenance period.Show more

This is a phase 3, prospective, open-label, single-arm, single-dose, multicenter study investigating the efficacy, safety, and tolerability of CSL222 (AAV5-hFIXco-Padua) in adolescent male participants with severe or moderately severe hemophilia B.

This study is a proof-of-concept phase 2b, multicenter, randomized, double-blind, placebo-controlled, parallel-group, dose selection study designed to explore the efficacy, safety, and tolerability of 2 active treatment regimens of CSL787 (human normal immunoglobulin G \[IgG\] inhalation solution) compared with placebo over 52 weeks. The primary aim of the study is to characterize the overall effect of CSL787 as well as the dose response of 2 active treatment regimens of inhaled CSL787 administered to participants with NCFB toward prolonging the time to first (TTF) exacerbation.Show more

This is a multicenter, parallel-group, double-blind, randomized phase 4 study designed to identify the optimal dose of CE1226 (2 active doses) to slow disease progression as assessed by reduced rates of annual lung density decline in alpha-1 antitrypsin (AAT) deficient participants over 3 years as compared with the marketed dose 60 milligrams per kilogram (mg/kg).Show more

This is a phase 2, randomized, multiple-dose, placebo-controlled study designed to evaluate the safety, efficacy, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and adolescents with sickle cell disease (SCD) experiencing vaso-occlusive crises (VOC). The main objectives of the study are to evaluate the safety and tolerability of CSL889 in study participants, and to assess how CSL889 affects the time it takes for VOC to resolve in participants with SCD.Show more

The purpose of this study is to assess the risk of bleeding due to failure of expected pharmacological action of CSL222 in adults with severe or moderately severe hemophilia B with detectable pretreatment AAV5 Nabs.

In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have not yet demonstrated long-term safety and effectiveness beyond the pivotal trials that led to their approval. In addition, results from well controlled, pivotal studies often cannot be replicated once a therapy has been approved for general use.2,3,4,5 In 2019 alone, the FDA has issued approvals for 24 new therapies for congenital and acquired hematologic conditions.6 In addition, almost 10,000 new studies for hematologic diseases are currently registered on www.clinicaltrials.gov.7 With this increase in potential new therapies possible, it is imperative that clinicians and clinical researchers in the field of non-neoplastic hematology have a uniform, secure, unbiased, and enduring method to collect long-term safety and efficacy data. As emphasized in a recently published review, accurate, uniform and quality national data collection is critical in clinical research, particularly for longitudinal cohort studies covering a lifetime of biologic risk.8Show more

This is a multinational, multicenter, prospective, observational cohort study of patients with HAE in the real-world setting. The study will include patients newly initiating garadacimab in routine clinical practice. Each participant will be followed for 48 months after index date (date of the first administration of garadacimab). Patient data will be collected from the HAE eDiary, patient medical records (MRs) and/or during a routine clinical visit and will be entered into the electronic case report form (eCRF) via an electronic data capture (EDC) system. Data pertaining to HAE attacks, prior HAE treatments, retrospective focused safety data collection, and healthcare resource utilization (HCRU) over a look-back period of 12 months prior to the enrollment will be extracted from the MR, and patients will also record retrospective HAE attack related data over a look-back period of 3 months prior to enrollment in the HAE eDiary. The primary aim of this study is to investigate the real-world effectiveness of garadacimab as measured by HAE attack rate before and after garadacimab initiation in patients with HAE over 24 months of follow-up. The study will aim to complement the data available from the clinical development program on the efficacy, safety, and health-related quality-of-life (HRQoL) in patients with HAE taking garadacimab.Show more

CSL Behring has 11 FDA-approved drugs with a rare disease use that we track, as of November 2026.

Hemgenix (etranacogene dezaparvovec), approved 2022: Hemophilia B.

Andembry (garadacimab), approved 2025: Hereditary Angioedema.

AFSTYLA Antihemophilic Factor (Recombinant), Single Chain (antihemophilic factor, human recombinant): Hemophilia A.

Berinert (human c1-esterase inhibitor): Hereditary Angioedema.

HAEGARDA C1 Esterase Inhibitor Subcutaneous (Human) (human c1-esterase inhibitor): Hereditary Angioedema.

Hizentra (human immunoglobulin g): Chronic Inflammatory Demyelinating Polyneuropathy, Common Variable Immunodeficiency, Wiskott-Aldrich Syndrome.

Humate-P (antihemophilic factor/von willebrand factor complex (human)): Hemophilia A, Von Willebrand Disease.

IDELVION (coagulation factor ix recombinant human): Hemophilia B.

And 3 more: see the FDA-approved drugs section on this page.

Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.

Yes, for 2 of its FDA-approved rare disease medicines that we track, each checked on CSL Behring's own website.

Hemgenix: HEMGENIX Connect, 1-833-436-0021. Offers copay help with private insurance, travel help and insurance help. Checked September 24, 2026.

Andembry: ANDEMBRY Connect, 1-844-423-4273. Offers copay help with private insurance, a short-term supply while insurance is decided and insurance help. Checked October 12, 2026.

Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.

Eligibility is decided by each program, so call the number listed before a prescription is filled.