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By PharmaCompass
2025-07-17
Impressions: 3,828 (Article) || 5 (Video)
This week’s Phispers brings you updates on cell and gene therapies (CGTs). After the death of a second teenaged boy last month who was on Elevidys for Duchenne muscular dystrophy (DMD), Sarepta Therapeutics has decided to undertake a restructuring exercise and is laying off 500 employees. Moreover, the US Food and Drug Administration (FDA) declined to approve Capricor Therapeutics’ lead cell therapy candidate for DMD, Deramiocel, and Ultragenyx’s gene therapy for a rare disease, UX111.
FDA Commissioner Marty Makary has said he wants to lower the PDUFA fee for the eighth iteration in order to reduce barriers before small companies, individual inventors and academia.
In deals, Ichnos Glenmark Innovation (IGI), the innovation arm of India’s Glenmark Pharmaceuticals, has signed an exclusive licensing agreement with AbbVie for its cancer treatment. IGI will receive an upfront payment of US$ 700 million and could earn up to US$ 1.225 billion in milestone payments. And Sino Biopharma has bought the remaining 95.09 percent stake in LaNova Medicines for US$ 950.92 million in order to enhance its oncology pipeline.
In approvals, FDA has granted full approval to Moderna’s Covid-19 vaccine, Spikevax, in children aged six months through 11 years who are at an increased risk of catching the disease. The agency also expanded the approval of Bayer’s chronic kidney disease drug Kerendia to include treatment of patients with two types of heart failure.
In trials, AstraZeneca’s experimental drug baxdrostat significantly lowered blood pressure in a phase 3 trial of patients with treatment-resistant hypertension. And the FDA has raised efficacy concerns over the use of Otsuka Pharma’s drug — brexpiprazole — in combination with Viatris’ Zoloft for the treatment of adults with post traumatic stress disorder (PTSD).
Post two deaths in Elevidys trial, Sarepta goes for restructuring, lays off 500 staffers
A month back, a second teenaged boy on Elevidys (delandistrogene moxeparvovec) had died in a clinical trial, post a similar death that had occurred in March. Elevidys is an FDA-approved gene therapy to treat DMD. Post this incident, Massachusetts-based Sarepta Therapeutics had suspended shipments of Elevidys for non-ambulant patients.
Now, the biotech has decided to let go of 500 employees (36 percent of its workforce) and is adding a black box warning for acute liver injury and acute liver failure to Elevidys, on instructions of the FDA. Sarepta is also halting the development of several gene therapies for a group of muscle wasting disorders, including those for limb-girdle muscular dystrophy. Through this strategic restructuring, Sarepta hopes to save US$ 400 million annually.
FDA rejects Capricor’s cell therapy: FDA has declined to approve Capricor Therapeutics’ Deramiocel, a lead cell therapy candidate for the treatment of cardiomyopathy associated with DMD. In its complete response letter (CRL), FDA said the evidence submitted for the therapy does not meet efficacy requirements and has asked for more data.
Rejects Ultragenyx’s gene therapy: FDA has declined to approve UX111, Ultragenyx’s gene therapy for Sanflilippo syndrome type A, a rare disease that causes progressive damage to the central nervous system. In the CRL, the agency has requested for additional information related to the company’s production processes and facilities.
FDA chief proposes lowering industry user fee to reduce barriers before small firms
The FDA has begun the reauthorization process for the eighth iteration of the Prescription Drug User Fee Act (PDUFA VIII), and Commissioner Marty Makary has said he wants to lower the PDUFA fees for this iteration of the program. “I’d like to see lower user fees. It’d be a reduced barrier for small companies and individual inventors and people in academics that may be trying to understand this process, including the capital requirement,” Makary said. The user fee program enables the agency to collect fees from companies that want to have their product applications reviewed.
Glenmark’s innovation arm signs licensing pact with AbbVie for its cancer drug
Ichnos Glenmark Innovation (IGI), the innovation arm of India’s Glenmark Pharmaceuticals, has signed an exclusive licensing agreement with AbbVie for its cancer treatment— ISB 2001. Under the agreement, AbbVie will get exclusive rights to develop, manufacture and commercialize ISB 2001 in North America, Europe, Japan and Greater China.
ISB 2001 is currently in phase 1 clinical trial in patients with relapsed or refractory multiple myeloma, a type of cancer that develops in white blood cells. IGI will receive an upfront payment of US$ 700 million and could earn up to US$ 1.225 billion in milestone payments.
Sino Biopharma buys out LaNova: After buying 4.91 percent stake in LaNova Medicines in November, Sino Biopharmaceutical is now acquiring the remaining 95.09 percent stake in the Chinese drugmaker for US$ 950.92 million. The acquisition will enhance Sino’s competitiveness and influence in oncology innovation.
Moderna gets full US approval for its Covid vaccine for at-risk infants, kids
The FDA has granted full approval to Moderna’s Covid-19 vaccine, Spikevax, in children aged six months through 11 years who are at an increased risk of the disease. This mRNA vaccine was previously available for pediatric populations under emergency use authorization. Moderna hopes to make it available for the eligible American population for the 2025-26 respiratory virus season.
Bayer’s Kerendia wins FDA label expansion: FDA has expanded the label of Bayer’s Kerendia (finerenone) to include treatment of patients with two types of heart failure. Kerendia can now be used for the treatment of adult patients with heart failure with either preserved ejection fraction or mildly reduced ejection fraction.
Concerns raised over Otsuka’s PTSD med: The FDA has raised efficacy concerns over the use of Otsuka Pharma’s drug — Rexulti (brexpiprazole) — in combination with Viatris’ Zoloft (sertraline) for the treatment of adults with PTSD. FDA has cited inconsistent trial results and a modest treatment effect and is insisting on another study on Rexulti. This assessment comes ahead of a meeting of independent experts to be held on July 18, on whether the agency should approve the med or not.
Astra’s BP drug from US$ 1.3 billion CinCor buyout, scores phase 3 win
AstraZeneca’s experimental drug baxdrostat significantly lowered blood pressure in a phase 3 trial on patients with treatment-resistant hypertension. The trial also successfully met all secondary endpoints. The company said it expects peak annual sales of the drug to exceed US$ 5 billion, driven by its use as a standalone therapy and in combination with Farxiga (dapagliflozin), its blockbuster treatment for type 2 diabetes, heart failure and chronic kidney disease. AstraZeneca had acquired baxdrostat through its 2023 purchase of CinCor Pharma for US$ 1.3 billion.
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