JAZZ PHARMACEUTICALS FRANCE

Ziihera, a dual HER2-targeted bispecific antibody, received accelerated FDA approval for HER2-positive biliary tract cancer (BTC) with a 52% objective response rate and median duration of response of 14.9 months. Zymeworks is eligible for up to $500M in regulatory milestones and $862.5M in commercial milestones. Regulatory reviews for zanidatamab are ongoing in China and Europe. The approval validates Zymeworks' Azymetric™ technology and capabilities in novel medicine development. Zanidatamab is also being investigated in Phase 3 trials for gastroesophageal adenocarcinomas and metastatic breast cancer.

FDA grants accelerated approval to Ziihera® (zanidatamab-hrii) for treating previously treated, unresectable or metastatic HER2-positive biliary tract cancer, based on a 52% objective response rate and 14.9-month median duration of response from the HERIZON-BTC-01 trial. Ziihera is the first dual HER2-targeted bispecific antibody approved for this indication in the U.S., with ongoing regulatory reviews in China and Europe. Zymeworks receives a $25M milestone payment from Jazz Pharmaceuticals and remains eligible for up to $500M in regulatory milestones.

Ziihera, a dual HER2-targeted bispecific antibody, received accelerated FDA approval for HER2-positive BTC, based on a 52% objective response rate and 14.9-month median duration of response. Zymeworks earned a $25M milestone payment and is eligible for up to $500M in regulatory milestones. Zanidatamab is also under regulatory review in China and Europe, and is being investigated in Phase 3 trials for gastroesophageal adenocarcinomas and metastatic breast cancer.

Early trials of antisense oligonucleotide (ASO) treatments for rare genetic epilepsies show promise in reducing seizures, but high costs pose challenges. ASOs have grown from a single N-of-1 study in 2019 to involve over two dozen patients, with some trials reporting significant seizure reductions. Despite promising results, the high cost of ASO treatments, potentially in the millions, raises concerns about accessibility. Neurologists emphasize the need for genetic screening at birth to identify patients early and streamline the development of these gene-based therapies.

Jazz Pharmaceuticals announced FDA accelerated approval of Ziihera 50mg/mL for injection for treating previously treated, unresectable or metastatic HER2-positive biliary tract cancer. Ziihera was approved based on a 52% objective response rate and a median duration of response of 14.9 months. Continued approval may depend on clinical benefit verification in the ongoing Phase 3 HERIZON-BTC-302 trial.

The FDA approved zanidatamab-hrii (Ziihera) for previously treated, unresectable, or metastatic HER2-positive biliary tract cancer, based on the phase 2b HERIZON-BTC-01 trial. The trial showed an objective response rate of 41.3% and median progression-free survival of 5.5 months.

Jazz Pharmaceuticals announces FDA accelerated approval of Ziihera, the first dual HER2-targeted bispecific antibody for HER2+ biliary tract cancer, based on a 52% objective response rate and 14.9-month median duration of response from the HERIZON-BTC-01 trial. Continued approval may depend on results from the ongoing Phase 3 HERIZON-BTC-302 trial.

Jazz Pharmaceuticals announces FDA approval of Ziihera, the first dual HER2-targeted bispecific antibody for HER2+ BTC, based on a 52% objective response rate and 14.9-month median duration of response from the HERIZON-BTC-01 trial.