Market Place
FDA okays BioMarin’s US$ 2.9 mn gene therapy for hemophilia A; Moderna forays into China with mRNA drugs
BioMarin Pharmaceutical’s one-time gene therapy – Roctavian – has been approved by the US Food and Drug Administration (FDA) to treat severe hemophilia A. Priced at US$ 2.9 million, Roctavian is the first gene replacement therapy to be approved for the condition.
In deals, Moderna has struck an agreement with Chinese officials to research, develop and manufacture messenger RNA medicines in China. Bausch + Lomb is set to acquire several eye-care products from Novartis through a potential US$ 2.5 billion deal. And Takeda has signed an immunotherapy deal with F-star Therapeutics.
The Centers for Disease Control and Prevention (CDC) has endorsed the use of Pfizer’s Abrysvo and GSK’s Arexvy to prevent RSV infections in older adults, making them the first vaccines against the disease in the US. Meanwhile, Moderna has taken its RSV vaccine to the FDA and other regulators to cash in on the multi-billion dollar opportunity. And, FDA has rejected Amneal Pharmaceuticals’ drug for Parkinson’s disease – IPX203 – citing insufficient safety data.
Several drugmakers like Boehringer Ingelheim, Sandoz, Organon and Samsung Bioepis, Coherus, Fresenius and Biocon Biologics have launched their biosimilars to AbbVie’s blockbuster drug Humira in the US this month.
AstraZeneca’s experimental lung cancer drug – datopotamab deruxtecan – has shown promise in slowing disease progression in a late-stage trial. And J&J and Protagonist Therapeutics have shared positive findings from a phase 2 trial on their experimental drug to treat moderate to severe plaque psoriasis.
FDA approves BioMarin’s US$ 2.9 million gene therapy for severe hemophilia A
BioMarin Pharmaceutical’s one-time gene therapy – Roctavian – has been approved by the FDA to treat severe hemophilia A, a rare bleeding disorder. Priced at US$ 2.9 million, Roctavian is the first gene replacement therapy to be approved for the condition.
Roctavian will compete with Roche’s antibody drug Hemlibra in the US. Meanwhile, FDA has also approved a companion diagnostic – AAV5 DetectCDx – to determine the most suitable patients eligible for the treatment. The test is the first of its kind to be cleared by the agency for use alongside a gene therapy.
Rejects Amneal’s Parkinson’s drug over safety: FDA has rejected Amneal Pharmaceuticals’ drug for Parkinson’s disease – IPX203 – citing insufficient safety data. The agency has expressed concerns about the safety of one of the drug’s ingredients and asked Amneal for more evidence.
Moderna signs MoU to research, develop, manufacture mRNA drugs in China
American vaccine maker Moderna said it is working towards opportunities to research, develop and manufacture mRNA medicines in China. To this effect, the drugmaker has signed a memorandum of understanding and a land collaboration agreement with the city government of Shanghai, China’s financial hub. According to a Reuters report, any medicines produced under this agreement will be exclusively for the Chinese people.
Bausch + Lomb to acquire Novartis eye products for potential US$ 2.5 billion
Bausch + Lomb has signed a potential US$ 2.5 billion deal with Novartis to acquire some of its eye-care products. The deal involves an upfront payment of US$ 1.75 billion from Bausch + Lomb, with potential milestone payments of up to US$ 750 million. The acquisition includes Novartis’ anti-inflammation eye drop Xiidra as well as the experimental drug Libvatrep for chronic ocular surface pain. The deal is expected to be finalized in the second half of 2023. Last year, Novartis had said it would focus on five major therapeutic areas – cardio-renal care, immunology, neuroscience, oncology and hematology.
Takeda in immunotherapy deal with F-star: F-star Therapeutics, an invoX company, has entered into a strategic discovery collaboration and licence agreement with Takeda. The collaboration will leverage F-star’s proprietary immunotherapies for patients with cancer. F-star will receive an undisclosed upfront payment as well as research funding and will also be eligible for milestone payments of up to US$ 1 billion, along with royalties.
CDC gives green light to Pfizer and GSK’s RSV vaccines for older adults
The CDC has endorsed the use of Pfizer’s Abrysvo and GSK’s Arexvy to prevent RSV infections in older adults, making them the first vaccines against the disease in the US. CDC said older adults may receive a single dose of a vaccine with the approval of their doctors. Pfizer and GSK are planning to supply the vaccines before this year’s RSV season.
Moderna takes its RSV jab to regulators: Moderna has started a rolling submission to the FDA for its RSV vaccine in adults 60 years and older. The drugmaker has also filed for regulatory review of its experimental RSV vaccine in Australia, Europe and Switzerland. With these submissions, Moderna hopes to take on Pfizer and GSK in the multibillion-dollar RSV market.
Several drugmakers launch Humira biosimilars in US: After Amgen became the first drugmaker to launch a biosimilar to AbbVie’s blockbuster drug Humira earlier this year, several drugmakers, like Boehringer Ingelheim, Sandoz, Celltrion Healthcare, Organon and Samsung Bioepis, Coherus, Fresenius and Biocon Biologics, have launched their biosimilars in the US this month. Boehringer’s Cyltezo has become the first and only FDA-approved interchangeable biosimilar to be launched in the US. Meanwhile, FDA has issued a complete response letter (CRL) to Alvotech’s Humira biosimilar, citing deficiencies at its manufacturing facility in Iceland.
Astra’s lung cancer drug shows promise in slowing disease progression
AstraZeneca’s experimental lung cancer drug – datopotamab deruxtecan – has shown promise in slowing disease progression in a late-stage trial. The drug, developed in collaboration with Daiichi Sankyo, demonstrated improved progression-free survival compared to standard chemotherapy in patients whose non-small cell lung cancer (NSCLC) had returned after one or two prior treatments.
The drugmaker, however, said some patients have died due to interstitial lung disease or scarring of lung tissue.
J&J, Protagonist share results from plaque psoriasis trial: Johnson & Johnson and biotechnology company Protagonist Therapeutics have shared findings from a phase 2 trial on their experimental drug to treat moderate to severe plaque psoriasis. After four months, a greater proportion of patients who received the drug instead of a placebo achieved at least a 75 percent reduction on a measure known as PASI, which assesses the size and severity of skin lesions. More drug recipients achieved 90 percent or total skin clearances as well, J&J said.
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