PharmaFocus Today – media

Early use of risdiplam in newborns with SMA shows improvements in motor development

Spinal muscular atrophy (SMA) is a rare genetic condition that causes progressive muscle weakness, which, when untreated, prevents infants with the most severe form from gaining motor development – never gaining the ability to sit – and typically leads to death before 2 years of age. The oral drug risdiplam benefits symptomatic patients with improved motor function and increased survival, but had only been Food and Drug Administration–approved for use in patients aged 2 months and older. An international consortium, co-led by Richard Finkel, MD, of St. Jude Children’s Research Hospital, formerly of Nemours Children’s Health, reports today that giving risdiplam as early as 16 days of age, before symptoms arise, is safe and effective. The phase 2 clinical trial results were published in the New England Journal of Medicine. “The impact of giving risdiplam soon after birth is quite dramatic,” said co-first and corresponding author Finkel, who is now the ...
- Source: drugdu
- 32
- August 14, 2025
FDA Approves First Drug for Chronic Lung Condition Bronchiectasis

WEDNESDAY, Aug. 13, 2025 (HealthDay News) — For the first time, people with bronchiectasis will have a treatment option. The U.S. Food and Drug Administration (FDA) has approved Insmed’s daily pill brensocatib, which will be sold under the brand name Brinsupri. The drug is designed to treat non-cystic fibrosis bronchiectasis (NCFB), a chronic condition that damages the lungs and makes it harder to clear mucus, the company said in a statement. The approval comes after the medication succeeded in one of last year’s most closely watched Phase 3 clinical trials. The company says Brinsupri could become a blockbuster, estimating peak sales at $5 billion a year, STAT News reported. “The FDA approval of the first-ever treatment for non-cystic fibrosis bronchiectasis is a historic milestone for patients and for Insmed,” the company’s chief medical officer, Dr. Martina Flammer, said in a statement. The American Lung Association estimates that bronchiectasis affects between ...
- Source: drugdu
- 32
- August 14, 2025
The scale of artificial intelligence applications in China is growing rapidly, with the average daily Token consumption exceeding 30 trillion

The Shanghai Securities News – China Securities Network (Reporter Yu Xiangming) Liu Liehong, a member of the Party Group of the National Development and Reform Commission and director of the National Data Administration, said at a series of theme press conferences on high-quality completion of the 14th Five-Year Plan held by the Information Office of The State Council on August 14 that the application scale of artificial intelligence in China is growing rapidly. At the beginning of 2024, the average daily consumption of tokens in China will be 100 billion. As of the end of June this year, the average daily consumption of tokens has exceeded 30 trillion, increasing by more than 300 times in one and a half years. The so-called Token, which is commonly referred to as a word element, serves as the smallest data unit for processing text, much like “traffic” in the Internet era. Liu Liehong ...
- Source: drugdu
- 26
- August 14, 2025
Novartis ‘ major new drug successfully completed Phase III

On August 11, Novartis announced that its anti-BAFF-R monoclonal antibody Ianalumab (VAY736) achieved the primary endpoint in two Phase III clinical trials in patients with Sjögren’s syndrome. Currently, there are no approved systemic therapies for Sjögren’s syndrome worldwide, and symptomatic treatments can only temporarily and partially relieve symptoms. Ianalumab is expected to end the history of Sjögren’s syndrome with no targeted drug available. Ianalumab is a novel, fully human monoclonal antibody targeting the B lymphocyte activating factor receptor (BAFF-R). It eliminates B cells through antibody-dependent cell-mediated cytotoxicity (ADCC) while simultaneously blocking BAFF-R-mediated B cell function and survival signals. The drug comes from Novartis’ partner Morphosys, which was acquired by Novartis for €2.7 billion in 2024. The drug is currently being developed for the treatment of a variety of B cell-driven autoimmune diseases, including Sjögren’s syndrome, immune thrombocytopenia (ITP), systemic lupus erythematosus (SLE), lupus nephritis (LN), warm antibody autoimmune hemolytic anemia ...
- Source: drugdu
- 26
- August 14, 2025
Hainan has issued policy measures to continuously increase support for the research and development of innovative drugs and medical devices

According to the website of the People’s Government of Hainan Province on August 14th, recently, the General Office of the People’s Government of Hainan Province issued the “Several Policy Measures of Hainan Province on Further Supporting the High-Quality Development of the Biomedical Industry”. The Policy Measures propose to continuously increase support for the research and development of innovative drugs and medical devices. Continue to implement the provincial biomedical industry research and development voucher policy. For the entire process of product research and development, including preclinical research, clinical trials, and industrialization of new achievements, provide financial rewards ranging from 400,000 to 10 million yuan based on phased achievements. The Policy Measures propose that enterprises both within and outside the province be encouraged to lawfully merge and restructure pharmaceutical enterprises in Hainan Province, revitalize assets on the spot and grow stronger. A 50% interest subsidy will be provided for bank loans arising ...
- Source: drugdu
- 26
- August 14, 2025
Freeze-dried Hib vaccine has been approved for clinical trials for the prevention of various invasive infections

Zhongzheng Intelligent Finance News Hualan Vaccine (301207) announced on the evening of August 13 that it has recently received the “Notice of Approval for Drug Clinical Trials” issued by the National Medical Products Administration for its freeze-dried Haemophilus influenzae type b conjugate vaccine (Hib vaccine). Agree to conduct clinical trials of this vaccine for the prevention of invasive infections caused by Haemophilus influenzae type b, including meningitis, pneumonia, sepsis, etc. It is reported that approximately 95% of invasive diseases caused by Haemophilus influenzae are triggered by type b strains. The Hib vaccine developed by the company is composed of purified Hib capsular polysaccharides combined with tetanus toxoid protein, and is in a freeze-dried dosage form. After vaccination, it can induce an immune response against Hib to provide protection. In addition, this vaccine is a component of the combined vaccine based on the component DPT that the company is developing. The ...
- Source: drugdu
- 25
- August 14, 2025
New medical insurance policy supports innovative drug CAR-T to bring breakthrough therapeutic effects and create diversified payment models

Recently, the National Healthcare Security Administration issued the “Several Measures to Support the High Quality Development of Innovative Drugs”, which clarifies the full chain support for the development of innovative drugs from research and development, admission, payment, etc. In this context, cell therapy, as a highly breakthrough innovative therapy in the field of biomedicine, is expected to further enhance the accessibility and affordability of its products. The Measures propose to encourage innovative drugs to conduct real-world research and link research results with medical insurance admission and renewal, promoting the application of real-world data in medical insurance admission. Of the 91 new drugs newly included in the medical insurance catalog in 2024, 33 were approved, launched, and included in the catalog that year, and innovative drugs are accelerating their integration into the medical insurance system. The clinical data accumulated through real-world research can more comprehensively verify the efficacy and safety of ...
- Source: drugdu
- 35
- August 13, 2025
Quadrivalent influenza vaccine Huierkangxin has been shortlisted for the initial review of the National Medical Insurance Innovation Catalogue.

Zhongzheng Smart Finance News: Zhuhai Zhuhui Biological – B (02627) announced in the morning of August 13 that according to the announcement of the National Healthcare Security Administration on August 12, the company’s quadrivalent influenza virus subunit vaccine, Huier Kangxin, has been included in the preliminary review list of innovative drugs for commercial health insurance in the country this year. It is the only vaccine product on the list. This vaccine was approved for marketing by the National Medical Products Administration in May 2023 and is suitable for people aged three and above (with a hemagglutinin concentration of 15μg/0.5ml for the virus strain). It is the first and only quadrivalent influenza virus subunit vaccine in China, providing broad protection against influenza viruses of type A H1N1, H3N2 and type B Yamagata and Victoria. The product is an optimized and upgraded version of traditional split vaccines, featuring comprehensive protection, high antigen ...
- Source: drugdu
- 35
- August 13, 2025
Roche terminates 12.2 billion yuan collaboration

Recently, Genentech, a subsidiary of Roche, announced the termination of its cooperation agreement with Bicycle Therapeutics. This decision will officially take effect in August. Genentech has had an exclusive partnership with Bicycle since February 2020, when Bicycle received a down payment of $30 million and is expected to receive a total transaction value of up to $1.7 billion (approximately RMB 12.2 billion) after Genentech reaches certain R&D milestones. However, the cooperation failed to proceed smoothly. Since 2023, Genentech has successively terminated several projects in the cooperation, until now it has completely stopped the cooperation. The reasons for the termination of the collaboration include, on the one hand, Genentech’s readjustment of project priorities and its gradual reduction of investment in Bicycle’s projects. On the other hand, Bicycle’s technology platform has certain limitations. Peptide screening is more difficult than antibody screening, and its shorter half-life limits development efficiency. Compared to antibody-drug conjugates ...
- Source: drugdu
- 35
- August 13, 2025
Hisense obtains approval to conduct clinical trials of HSK47977 tablets

August 12, Hisense(002653) announced that the company recently received the “Notice of Approval for Drug Clinical Trial” issued by the National Medical Products Administration, approving its independently developed innovative drugHSK47977 tablets are undergoing clinical trials. This drug is an oral BCL6 PROTAC small molecule formulation, primarily used to treat non-Hodgkin’s lymphoma, and meets the relevant drug registration requirements. HSK47977 tablets have no clinically active drugs targeting the same target in China, and they hold the potential to become a first-in-class product. The company completed Pre-IND discussions with the FDA in July 2025 and submitted an IND application, with the potential for simultaneous development in China and the US. Preclinical studies have demonstrated potent anti-tumor activity and a favorable safety profile, demonstrating high development potential. In the first quarter of 2025, Hisense achieved revenue of 892 million yuan and net profit attributable to shareholders of the parent company of 46.61 million ...
- Source: drugdu
- 29
- August 13, 2025

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