EMA Approves TAKHZYRO for Additional Subcutaneous Administration Option to Treat Hereditary Angioedema

The European Medicines Agency (EMA) has approved Takeda’s lanadelumab (brand name TAKHZYRO) in a 2-mL pre-filled pen as a subcutaneous administration option for treating hereditary angioedema (HAE) in adolescents 12 years and above and adult patients, Takeda announced on Feb. 24, 2025 (1).

This approval is for a 300-mg solution for injection in pre-filled pen, which contains 300 mg of lanadelumab in 2 mL of solution. The approval was supported by a clinical study (2). Lanadelumab was previously approved as a 150-mg solution and a 300-mg solution for injection in pre-filled syringe and as a 300-mg solution for injection in vial (3).

“HAE affects an estimated 1 in 50,000 people worldwide and is often under recognized, under diagnosed, and under treated [4]. We welcome the swift approval by the EMA on this additional subcutaneous administration option,” said Irmgard Andresen, global medical lead HAE at Takeda, in a company press release (1). “HAE patients 12 years and older now have an additional individualized treatment option available to them.”

HAE is a rare genetic disorder that results in recurring attacks of oedema, or swelling, in various parts of the body. The swelling can occur in the abdomen, face, feet, genitals, hands, and throat, and can be debilitating and painful (5). Furthermore, attacks that obstruct airways can lead to asphyxiation and thus are potentially life threatening (6).

Lanadelumab is a fully human monoclonal antibody that binds specifically to plasma kallikrein and decreases its levels. It has consistently demonstrated reduction in attacks of HAE.

In earlier news, Takeda received approval from the Japanese Ministry of Health, Labour and Welfare (MHLW) in late December 2024 for HYQVIA [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] for treating patients with agammaglobulinemia or hypogammaglobulinemia. Patients suffering from these disorders have very low or absent levels of antibodies and carry an increased risk of serious recurring infection caused by primary immunodeficiency or secondary immunodeficiency. HYQVIA’s approval in Japan makes it the first and only facilitated subcutaneous immunoglobulin (fSCIG) therapy available for patients in that country.

“There is a high unmet need for plasma-derived therapies (PDTs) in patients in Japan, which is anticipated to increase as education and timely diagnosis rates continue to improve,” said Kristina Allikmets, head of R&D for Takeda’s Plasma-Derived Therapies Business Unit, in a company press release issued at the time of approval (7).

“The approval of HYQVIA, the first and only facilitated SCIG treatment, is further evidence of Takeda’s commitment to add to the standard of care for patients in Japan. We look forward to continuing to bring new therapeutic options that support and enhance the experience of patients in our home country throughout the next decade,” Allikmets stated in the release.

Meanwhile, Takeda also announced in March 2025 that a Phase III study of rusfertide, an investigational hepcidin mimetic peptide therapeutic, met its primary endpoint in phlebotomy-dependent patients with polycythemia vera (PV) (8). According to a company press release, “[p]atients with PV are at increased risk for life-threatening cardiovascular and thrombotic events. Many patients with PV require regular phlebotomy, a process of removing blood to manage elevated hematocrit levels caused by an excess of red blood cells, as well as treatment with cytoreductive therapies” (8).

References

1. Takeda. The European Medicines Agency (EMA) Has Approved an Additional Subcutaneous Administration Option for TAKHZYRO (lanadelumab) for Patients Aged 12 Years and Above with Recurrent Attacks of Hereditary Angioedema (HAE). Press Release. Feb. 24, 2025.
2. ClinicalTrials.gov. A Study to Determine the Bioavailability of Lanadelumab (SHP643) Administered Subcutaneously with the Prefilled Syringe and the Autoinjector in Healthy Adult Volunteer Participants. clinicaltrials.gov (accessed March 6, 2025).
3. EMA. Takhzyro. ema.europa.eu (accessed March 6, 2025).
4. Longhurst, H. J.; Bork, K. Hereditary Angioedema: An Update on Causes, Manifestations and Treatment. Br. J. Hosp. Med. 2019, 80 (7), 391–398.
5. Maurer, M.; Magerl, M.; Betschel, S.; et al. The International WAO/EAACI Guideline for the Management of Hereditary Angioedema—The 2021 Revision and Update. Allergy 2022, 77 (7), 1961–1990. DOI: 10.1111/all.15214
6. Banerji, A.; Davis, K. H.; Brown, T. M.; et al. Patient-reported Burden of Hereditary Angioedema: Findings from a Patient Survey in the United States. Ann. Allergy, Asthma, Immunol. 2020, 124 (6), 600–607.
7. Takeda. Takeda Announces Approval of HYQVIA 10% S.C. (Subcutaneous) Injection Set in Japan for Patients with Agammaglobulinemia or Hypogammaglobulinemia. Press Release. Dec. 27, 2024.
8. Takeda. Protagonist and Takeda Announce Positive Topline Results from Phase 3 VERIFY Study of Rusfertide in Patients with Polycythemia Vera. Press Release. March 3, 2025.