Nog even en ook de verkopen in China zullen op gang komen. Argenx heeft significante royalties op de verkoopscijfers die partner Zai Lab in China realiseert. Zie PR van 6 januari 2021:
argenx is also eligible to receive tiered royalties (mid-teen to lowtwenties on a percentage basis) based on annual net sales of efgartigimod in Greater China.
Sep 18, 2023
Zai Lab Obtains Breakthrough Therapy Designation for Efgartigimod Alfa Injection (Subcutaneous Injection) in Patients with Chronic Inflammatory Demyelinating Polyneuropathy in China
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SHANGHAI, China and CAMBRIDGE, Mass., Sept. 18, 2023 (GLOBE NEWSWIRE) -- Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted Breakthrough Therapy Designation for efgartigimod alfa injection (subcutaneous injection) (efgartigimod SC) for the treatment of patients with chronic inflammatory demyelinating polyneuropathy (CIDP). The Breakthrough Therapy Designation for efgartigimod SC was supported by data from both global and Chinese patients enrolled in the ADHERE study.
“In granting Breakthrough Therapy Designation, we are pleased to see that the CDE recognizes the promise of efgartigimod SC as an innovative treatment option for CIDP patients,” said Dr. Harald Reinhart, President and Head of Global Development, Neuroscience, Autoimmune & Infectious Diseases, Zai Lab. “We have seen how efgartigimod SC can meaningfully improve and stabilize disease symptoms in these patients. Existing treatment options are quite limited, and problematic given the general reliance on long-term steroid or chronic immunoglobulin therapy. In the ADHERE study, significant efficacy was demonstrated with a favorable safety profile. We are excited about the therapeutic potential of efgartigimod SC in CIDP, and we look forward to working with regulatory authorities in China to bring this important medicine to patients in need as soon as possible.”
The Breakthrough Therapy Designation review policy is designed to facilitate the development and expeditious review of novel medicines that are intended for the prevention or treatment of serious, life-threatening diseases or diseases that severely impact the quality of life for which there is no existing treatment, or where sufficient evidence indicates advantages of the novel drug over currently available treatment options. Drugs granted Breakthrough Therapy Designation receive priority communications and guidance from the CDE to promote and expedite the drug review process.
In July 2023, Zai Lab and argenx reported positive topline results from the ADHERE study evaluating efgartigimod SC in adults with CIDP.
Primary endpoint met (p=0.000039); efgartigimod SC demonstrated 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in the risk of relapse versus placebo.
67% of global study participants in open-label Stage A demonstrated evidence of clinical improvement (ECI) indicating that IgG autoantibodies play a significant role in the underlying biology of CIDP.
Safety and tolerability profile consistent with confirmed safety profile of VYVGART.
In the subgroup analysis for ADHERE trial participants in China, the results were consistent with global outcomes.
The subgroup analysis of the mainland Chinese participants demonstrated an impressive reduction in relapse risk by 69% with efgartigimod SC compared to placebo.
The mainland Chinese participants showed a similar level of response compared to the global population, with 78% of the mainland Chinese participants treated with open-label efgartigimod SC demonstrating confirmed ECI.
In the mainland Chinese subgroup, efgartigimod SC demonstrated a safety profile consistent with that observed in the global population.
About ADHERE Trial Design
The ADHERE trial, sponsored by argenx, was a multicenter, randomized, double-blind, placebo-controlled trial evaluating efgartigimod SC for the treatment of CIDP. ADHERE enrolled 322 adult patients with CIDP who were treatment naïve (not on active treatment for =6 months) or being treated with immunoglobulin therapy or corticosteroids. Zai Lab enrolled patients in the ADHERE trial in Greater China (mainland China, Hong Kong, Taiwan and Macau). The trial consisted of an open-label Stage A followed by a randomized, placebo-controlled Stage B. In order to enter Stage A and receive efgartigimod SC the diagnosis of CIDP was confirmed by an independent panel of experts. Patients entered a run-in stage, where any ongoing CIDP treatment was stopped and they had to demonstrate active disease, with clinically meaningful worsening on at least one CIDP clinical assessment tool, including INCAT, I-RODS, or mean grip strength. Treatment naïve patients were able to skip the run-in period with proof of recent worsening. To advance to Stage B, patients needed to demonstrate ECI to efgartigimod SC. ECI was achieved through improvement of INCAT score, or improvement on I-RODS or mean grip strength if those scales had demonstrated worsening during the run-in period. In Stage B, patients were randomized to either efgartigimod SC or placebo for up to 48 weeks. The primary endpoint was based on the hazard ratio for the time to first adjusted INCAT deterioration (i.e. relapse). After Stage B, all patients had the option to roll-over to an open-label extension study to receive efgartigimod SC.
About CIDP in China
The prevalence of CIDP in China is estimated at 50,000 patients.1 Current treatment options are primarily corticosteroids and intravenous immunoglobulin (IVIg), with plasma exchange (PLEX) generally reserved for refractory patients. There is limited access to PLEX or IVIg in many parts of the world, including China. As most patients require treatment for an extended period of time there remains a significant unmet need for alternate treatment options that are effective, well-tolerated, and convenient for patients with CIDP in China.
1 Chronic inflammatory demyelinating polyneuropathy and diabetes, 2020.
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