FDA Approves AstraZeneca and Ionis' Wainua for Hereditary ATTR Polyneuropathy
核心洞察
The FDA has approved AstraZeneca and Ionis' Wainua (eplontersen) for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN (搜索)).
Approval was based on the NEURO-TTRansform study, demonstrating Wainua's ability to improve neuropathy impairment and quality of life.
Merck (搜索) received a Complete Response Letter (CRL) from the FDA for gefapixant, its chronic cough (搜索) candidate, marking the second CRL for this drug.
The FDA has granted approval to Wainua (eplontersen), developed by AstraZeneca and Ionis Pharmaceuticals, for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN (搜索)) in adults. This debilitating condition affects approximately 40,000 people worldwide, leading to progressive peripheral nerve damage.
The approval of eplontersen, to be marketed as Wainua, is based on data from the pivotal NEURO-TTRansform study. The results demonstrated a consistent and sustained benefit in patients treated with Wainua, showing significant improvements in neuropathy impairment and overall quality of life. Wainua is anticipated to be available in the United States starting January 2024. Applications for eplontersen's approval for ATTRv-PN (搜索) are currently under review in the European Union.
Clinical Trial Data
The NEURO-TTRansform study evaluated the efficacy and safety of eplontersen in patients with ATTRv-PN (搜索). Key findings included statistically significant improvements in the modified Neuropathy Impairment Score +7 (mNIS+7) and the Norfolk Quality of Life-Diabetic Neuropathy (QoL-DN) questionnaire. These results underscore Wainua's potential to address both the neurological and quality-of-life aspects of ATTRv-PN.
AstraZeneca and Ionis are also investigating eplontersen for cardiomyopathy caused by hereditary TTR amyloidosis in the Phase III CARDIO-TTRansform study, with data expected in the first half of 2025.
Merck's Gefapixant Receives Second CRL
In other regulatory news, Merck (搜索) has received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for gefapixant, a candidate for treating refractory or unexplained chronic cough (搜索). This marks the second CRL issued for gefapixant.
The FDA's latest CRL indicated that the NDA did not provide substantial evidence of gefapixant's effectiveness in treating refractory or unexplained chronic cough (搜索). Merck (搜索) clarified that the CRL was not related to safety concerns.
Keytruda Gains European Approval for Gastrointestinal Cancers
Meanwhile, the European Commission has approved Keytruda for two indications in gastrointestinal cancers. The approvals cover Keytruda in combination with gemcitabine and cisplatin as a first-line treatment for locally advanced unresectable or metastatic biliary tract cancer (搜索), based on the Phase III KEYNOTE-966 study. Additionally, Keytruda plus fluoropyrimidine- and platinum-containing chemotherapy was approved for first-line treatment of locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma, based on the KEYNOTE-859 study. Keytruda is now approved for seven gastrointestinal cancers in Europe.