By Heather McKenzie https://www.biospace.com/ Pictured: Sarepta logo on a building/courtesy of Sarepta Therapeutics Those in the Duchenne muscular dystrophy and gene therapy spaces hoping for a milestone decision by May 29 will have to wait approximately three more weeks as the FDA set a new action date of June 22 for Sarepta’s gene therapy for the neuromuscular disease. The FDA told the company Wednesday “it requires modest additional time to complete the review, including final label negotiations and postmarketing commitment discussions,” according to Sarepta’s announcement. The company went on to say that the FDA indicated it is “working toward potentially granting an accelerated approval for SRP-9001” for DMD patients aged 4-5 years. If approved, SRP-900 would be the first FDA-authorized gene therapy for DMD. Sarepta’s shares were down 14% in pre-market trading Wednesday.
By Jef Akst Pictured: Illustration of a human body with the intestines highlighted/iStock, Rasi Bhadramani At $17 per share, Ironwood Pharmaceuticals has agreed to buy VectivBio for around $1 billion in a cash deal. In doing so, the Boston-based company, which already markets Linzess for irritable bowel syndrome and constipation with AbbVie, will expand its gastrointestinal drug portfolio. The Swiss biotech’s most advanced product is apraglutide, being developed for short bowel syndrome with intestinal failure (SBS-IF) that affects some 18,000 adult patients in the U.S., Europe and Japan, according to the Ironwood press release. People with the disorder are unable to absorb adequate nutrition from their food, leading to impaired quality of life and sometimes death. Takeda Pharmaceuticals has an approved drug, Gattex, that aims to treat short bowel syndrome but requires daily injections. Apraglutide is being tested in a Phase III study involving weekly injections. “There’s certainly challenges with ...
AbbVie and Genmab’s blood cancer therapy, Epkinly (epcoritamab-bysp), has been granted accelerated approval by the US Food and Drug Administration (FDA). The authorisation specifically applies to adults with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) who have received at least two prior lines of treatment. DLBCL is the most common type of aggressive lymphoma, with approximately 150,000 cases of the disease diagnosed globally each year. For R/R patients, several targeted therapies including T-cell mediated treatments have recently emerged, the companies said. However, single agent and ready-available or off-the-shelf treatment options are limited. Epkinly, which is being co-developed by AbbVie and Genmab as part of the companies’ oncology collaboration, is now the first and only T-cell engaging bispecific antibody for this indication. The FDA’s decision was supported by positive results from a phase 1/2 trial, in which Epkinly delivered an overall response rate of 61%, a complete response rate ...
By Tristan Manalac Pictured: Blue sign outside FDA building/JHVEPhoto/Adobe Stock The FDA has approved Krystal Biotech’s topical gene therapy beremagene geperpavec-svdt, now to be marketed as Vyjuvek, for the treatment of the rare skin disease dystrophic epidermolysis bullosa, the company announced Friday. Vyjuvek’s label covers its use in dystrophic epidermolysis bullosa (DEB) patients aged six months and above bearing mutations in the COL7A1 gene. Friday’s approval makes Vyjuvek therapy the first-ever redosable gene therapy and the first DEB therapeutic to hit the market. “Until now, there have been no approved treatment for DEB in the United States, and patients, families and physicians were limited to palliative care,” Andy Orth, chief commercial officer at Krystal Biotech, said in an investor call Friday evening, adding that there are likely around 3,000 DEB patients in the U.S., of whom some 1,100 have confirmed diagnoses, according to claims analytics. The company expects to launch ...
The therapy is a brain-penetrant used in various combinations in patients with progressive glioblastoma Debiopharm – a company focusing on future standards of cancer care and infectious diseases – has announced the beginning of its open-label phase 1/2 research of its candidate Debio 0123. The therapy is a brain-penetrant and selective WEE1 inhibitor, used in combination with temozolomide (TMZ) in patients with recurrent or progressive glioblastoma (GBM). It is also being studied in combination with TMZ/RT (SOC) in newly diagnosed patients. The commencement phase of the clinical study aims to establish the recommended phase 2 doses of the treatment. Ultimately, Debiopharm is aiming to meet the pressing requirement for novel treatment interventions to boost quality of life and clinical outcomes for patients living with GBM. Among cancer cells, DNA damage response (DDR) pathways are often upregulated due to genomic instability, increasing the chances of resistance to DNA-damaging therapies. Blocking DNA ...
Mustang Bio is galloping away from a few City of Hope-partnered cell therapy programs and pausing a gene therapy trial to funnel resources toward a lead asset developed in collaboration with Fred Hutch. A pipeline reprioritization has led the company to ax work on a CD123-targeted CAR T as well as three other cell therapies targeting HER2, CS1 and PCSA, respectively, according to a release Thursday. Mustang will continue work on an IL13Rα2-targeting cell therapy combined with an oncolytic virus—MB-108—that’s been developed alongside City of Hope and Nationwide Children’s Hospital. And that’s not all; Mustang is halting “bubble boy disease” gene therapy after a review of data from an ongoing investigator-sponsored trial spurred a pause in enrollment. The company says no safety concerns have been raised and “no insertional mutagenesis or malignancy has been detected” in either investigator-sponsored trial. Mustang is awaiting more data from its partner on a new ...
With scarce clinical resources unable to keep pace with elevated rates of mental illness, technological solutions could help decrease waitlists and disparities in access to therapy. Recent advances in artificial intelligence have fueled interest in the use of chatbots and virtual assistants for mental health treatment. A new study led by researchers from University of Illinois Chicago, with collaborators at Washington University and Pennsylvania State University, finds that an AI voice assistant app delivering a form of psychotherapy can help patients with mild depression and anxiety. The article, published in Translational Psychiatry, reports changes in brain activity along with improved depression and anxiety symptoms after using the AI voice assistant, called Lumen, for eight sessions of problem-solving therapy. The results of this pilot study, the first to test an AI voice-based virtual coach for behavioral therapy, offer encouraging evidence that virtual therapy can play a role in filling the gaps ...
A US Food and Drug Administration’s (FDA) panel of experts has recommended Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) investigational gene therapy for accelerated approval. The Cellular, Tissue and Gene Therapies advisory committee voted eight to six in support of SRP-9001 (delandistrogene moxeparvovec) as a treatment for ambulatory DMD patients with a confirmed mutation in the DMD gene. DMD is a rare and inherited X-chromosome-linked disease that results in the lack of dystrophin protein, which is required to strengthen and protect muscles. Over time, this causes progressive loss of muscle strength, with most patients requiring full-time use of a wheelchair by their early teens. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The disease almost exclusively affects males, occurring in approximately one in every 3,500-5,000 newborn males worldwide. SRP-9001 is designed to address the underlying cause of DMD ...
Reviewed by Lily Ramsey, LLM Investigators at Brigham and Women’s Hospital and collaborating institutions evaluated the dual-therapeutic effect of gene therapy in a clinically relevant model for common form of bone cancer. With a worldwide incident rate of 3.4 cases per million people per year, osteosarcoma is one of the most common bone cancers affecting children and adolescents. The current gold standard treatment option requires extensive surgical intervention and chemotherapy that leads to a poor prognosis and decreased quality of life. Due to the aggressive nature of the disease, the surgical intervention usually involves total reconstruction of the limbs or, in most cases, amputation. Researchers at Brigham and Women’s Hospital, a founding member of the Mass General Brigham healthcare system, in collaboration with investigators at University College Dublin (UCD), Massachusetts Institute of Technology (MIT), and Trinity College Dublin (TCD), have identified a potential therapeutic target and developed a unique delivery ...
In the first year of Covid-19, Medicare paid providers $1 billion for psychotherapy services, and $580 million were improper payments, including $348 million that were for telehealth services, a recent audit by the Office of Inspector General (OIG) found. The OIG audit, published Tuesday, analyzed $1 billion in Part B payments for more than 13.5 million psychotherapy services from March 2020 through February 2021. Its analysis consisted of two random samples of psychotherapy services: a sample of 111 enrollee days for telehealth services and a sample of 105 enrollee days for non-telehealth services. An enrollee day includes “all claim lines for Medicare Part B psychotherapy services with the same service start date for a specific enrollee,” the OIG said. Audits from before the pandemic showed improper payments for psychotherapy services, leading the agency to examine payments during the pandemic when telehealth access was greatly expanded. “Because of the significant increase ...
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