AstraZeneca Announces China Approval of Koselugo® (Selumetinib) for Pediatric and Adult Patients with Symptomatic, Inoperable Plexiform Neurofibromas in Neurofibromatosis Type 1

AstraZeneca Announces Formal Approval of Cosentyx by China’s National Medical Products Administration^®(Selumetinib Sulfate Capsules), a selective, oral MEK inhibitor, indicated for the treatment of pediatric and adult patients aged 3 years and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN)¹。

This approval is based on the positive results from the Phase III KOMET clinical trial, which is the largest and only global, multicenter, randomized, placebo-controlled Phase III clinical trial conducted in adult patients with NF1-PN.

On March 28, 2026, Chinese patient data from the KOMET study were released at the Neurofibromatosis Special Disease Forum during Shanghai Plastic Surgery Technology Week. Subgroup analysis of Chinese patients showed that the objective response rate (ORR) reached 54.5% by the 16th treatment cycle, demonstrating significant clinical benefit compared with placebo.

NF1 is a rare, progressive, genetic disorder that is typically diagnosed in early childhood, often persists into adulthood, and can lead to multisystem involvement throughout the body.^3,4Up to 50% of patients with neurofibromatosis type 1 (NF1) develop plexiform neurofibromas (PN), which can involve the brain, spinal cord, and peripheral nervous system. PN may also manifest later in life and continue to grow in size, leading to various disabling symptoms such as pain, deformity, and muscle weakness.^4,5。

Sun Yat-sen University Cancer Center, Professor Chen Zhongping, Lead Investigator of the KOMET Study in China"Adult patients with NF1-associated plexiform neurofibromas (NF1-PN) often face a substantial clinical burden. Real-world experience with selumetinib in pediatric patients has firmly established its efficacy in children with NF1-PN. As the first global Phase III clinical trial conducted specifically in adult patients with symptomatic, inoperable NF1-PN, the KOMET study further validated that selumetinib significantly controls tumor progression and improves clinical symptoms, while demonstrating a favorable safety profile. Notably, four research centers in China contributed high-quality data to the KOMET study, providing critical support for these global findings. This progress holds promise for comprehensive, lifelong management of NF1-PN from childhood through adulthood, and we look forward to this innovative therapy benefiting more patients in the near future."

Shenzhen Paopao Home Neurofibromatosis Care CenterTransmissionDepartment Director Zou YangIndicate：“For adult patients with NF1, long-term disease progression often leads to a substantial disease burden, while existing treatment options remain very limited, leaving a significant unmet need. Selumetinib has previously demonstrated clear clinical benefits in pediatric patients, and this expansion of indications brings long-awaited hope to the adult patient population. We are truly delighted for these patients. Meanwhile, this important milestone fully demonstrates the power of collaborative efforts among industry partners, clinicians, and patient organizations to illuminate new possibilities for those with rare diseases.”

He Jing, Senior Vice President of AstraZeneca Global and Head of Global R&D ChinaPh.D.“Scientific innovation in the field of rare disease treatment is a key focus of AstraZeneca’s R&D strategy. The successful approval of selumetinib for the adult indication of neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) was made possible by the active participation of Chinese researchers. Supported by data from the pivotal Phase III KOMET clinical trial, this approval provides a new treatment option for adult patients with this condition in China, facilitating comprehensive lifecycle management from pediatric to adult patients. Moving forward, we will continue to address unmet needs in the rare disease space, accelerate the introduction of innovative medicines, and conduct local research, thereby extending the benefits of science to a broader population of individuals with rare diseases.”

Hu Yiqing, Vice President of AstraZeneca China and Head of the Rare Disease Business Unit, stated:“AstraZeneca remains committed to addressing the challenges faced by patients with rare diseases, leveraging scientific innovation to bring new hope to Chinese patients and help them regain their roles as pillars of society. From childhood to adulthood, selumetinib covers the full spectrum of treatment needs for patients with NF1, offering adult patients the potential to control disease progression and alleviate symptoms. It also holds promise for reducing the caregiving burden on families, enabling patients to better return to normal life, integrate into society, and realize their self-worth.”

The study showed that, in the primary analysis of the Phase III KOMET clinical trial, the selumetinib group demonstrated a statistically significant and clinically meaningful objective response rate (ORR) compared with the placebo group.

In the KOMET Phase III clinical study, the safety profile of selumetinib was consistent with its previously known safety profile and prior experience in pediatric patients.³。

Based on the results of the Phase III KOMET clinical trial, selumetinib has been approved in the United States, the European Union, Japan, and other countries for the treatment of adult patients with symptomatic, unresectable NF1-associated plexiform neurofibromas (NF1-PN). Regulatory reviews in other countries/regions are ongoing.

*As ofMarch 30, 2026，The first and only one in mainland China.

This article involves products or indications that have not yet been approved in mainland China. AstraZeneca does not recommend the use of any unapproved medicines.

Neurofibromatosis Type 1 (NF1) is a rare, progressive, genetic disorder caused by spontaneous or inherited mutations in the NF1 gene.^3,4. NF1 can be associated with a variety of clinical manifestations, including soft masses on the skin surface and subcutaneously (cutaneous neurofibromas). Additionally, in up to 50% of patients, tumors known as plexiform neurofibromas (PN) may form at the nerve sheath.^4,5. These PN can cause various clinical problems, including cosmetic deformities, motor dysfunction, pain, airway dysfunction, visual impairment, and bladder or bowel dysfunction.^4,5。

KOMET is a global, Phase III, randomized, double-blind, placebo-controlled, multicenter clinical study designed to evaluate the efficacy and safety of selumetinib in adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PNs). The study enrolled 145 adult patients from 13 countries across North America, South America, Europe, Asia, and Australia. Their baseline characteristics, including sex and PN distribution, are representative of the global adult NF1 patient population. Patients were randomized in a 1:1 ratio to receive either selumetinib or placebo for 12 cycles, each lasting 28 days. Eligible patients were required to have a confirmed diagnosis of NF1, at least one symptomatic, inoperable PN measurable by volumetric MRI analysis, documented chronic PN pain scores during the screening period, adequate organ and bone marrow function, and stable use of medications for chronic PN pain at enrollment.^6,7。

The primary endpoint was the objective response rate (ORR) at treatment cycle 16, as confirmed by independent central review (ICR). ORR was defined as the proportion of patients achieving a confirmed complete response (disappearance of plexiform neurofibromas [PN]) or partial response (at least a 20% reduction in tumor volume from baseline). Secondary endpoints included improvement in PN-related pain and improvement in scores on the PN-related quality of life assessment scale (PlexiQoL) at treatment cycle 12.^6,7。

Upon completion of 12 treatment cycles, patients initially receiving placebo were switched to selumetinib therapy, while those initially receiving selumetinib continued for an additional 12 treatment cycles. Patients who completed 24 treatment cycles could opt to enter the long-term extension phase and continue receiving selumetinib therapy.^6,7。

Selumetinib is a kinase inhibitor that blocks specific enzymes (MEK1 and MEK2) involved in the process of stimulating cell growth. In NF1, the activity of these enzymes is abnormally enhanced, leading to unregulated growth of tumor cells and the formation of so-called plexiform neurofibromas (PN). By blocking the activity of these enzymes, selumetinib can slow the growth of tumor cells, thereby slowing the growth of PN.

Selumetinib has been approved in the United States, the European Union, Japan, China, and other countries/regions for the treatment of pediatric patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

Selumetinib has been approved in the United States, the European Union, Japan, China, and other countries/regions for the treatment of adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PNs). Regulatory reviews are still ongoing in other countries/regions.

Selumetinib has received orphan drug designation for the treatment of NF1 in the United States, the European Union, Japan, and other countries.

As a subsidiary of AstraZeneca’s Rare Disease business, Alexion Pharmaceuticals is dedicated to researching, developing, and providing life-transforming medicines for patients with rare diseases and their families. More than three decades ago, Alexion pioneered the translation of the complex biology of the complement system into transformative therapies and has continued to drive robust research and development innovation in disease areas with significant unmet needs. Leveraging AstraZeneca’s global resources, Alexion is continuously expanding its footprint to serve more patients with rare diseases worldwide. Alexion Pharmaceuticals is headquartered in Boston, Massachusetts, USA.

AstraZeneca (LSE/STO/NYSE: AZN) is a science-led global biopharmaceutical company dedicated to the research, development, manufacturing, and marketing of prescription medicines, with a strategic focus on oncology, rare diseases, and biopharmaceuticals including cardiovascular, renal, and metabolism; respiratory; and immunology. Headquartered in Cambridge, United Kingdom, AstraZeneca operates in more than 125 countries, delivering innovative medicines that benefit millions of patients worldwide.

References

1.Koselugo (selumetinib). Chinese prescribing information; 2026.

2.The 15th International Symposium on Plastic & Cosmetic Surgery / The 25th International Congress of Plastic & Reconstructive Surgery in Shanghai, 2026.    

3.Tamura R. Current understanding of neurofibromatosis type 1, 2, and schwannomatosis. Int J Mol Sci. 2021;22(11):5850.

4.Hirbe AC, et al. Neurofibromatosis type 1: a multidisciplinary approach to care. Lancet Neurol. 2014;13:834-843.

5.Bergqvist C, et al. Neurofibromatosis 1 French national guidelines based on an extensive literature review since 1966. Orphanet J Rare Dis. 2020;15(1):37.

6.Chen, AP, et al. KOMET: a phase 3, multicentre, international, randomised, placebo-controlled study to assess the efficacy and safety of selumetinib in adults with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas. The Lancet. 2025;405(10496):2217-2230.

7.ClinicalTrials.gov. Efficacy and safety of selumetinib in adults with NF1 who have symptomatic, inoperable plexiform neurofibromas (KOMET). NCT Identifier: NCT04924608. Available here. Accessed March 2026.