Search Results for “FDA” – Page 29 – media

Vertex, CRISPR score landmark FDA approval for sickle cell disease gene therapy Casgevy

Groundbreaking? Game-changing? Transformational? Historic? None of the buzzwords sound adequate to describe Friday’s FDA approval of Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exa-cel) to treat sickle cell disease (SCD). The therapy is a long-awaited potential cure for the debilitating and life-threatening disease which affects more than 100,000 in the United States, most of them Black. It’s also the first medicine using the revolutionary CRISPR gene-editing system, which earned its inventors a Nobel Prize in 2020 and holds tantalizing potential to cure diseases for which there is no treatment. Friday, in surprising timing, the FDA approved a second gene therapy for SCD, bluebird bio’s Lyfgenia (lovo-cel), which was due for a decision Dec. 20. Both approvals are for people 12 and older. The FDA has signed off on roughly two dozen cell or gene therapies, beginning with Novartis’ Kymriah in 2017, but none have the potential impact of Casgevy and Lyfgenia. ...
- Source: drugdu
- 111
- December 12, 2023
FDA Approves Two Landmark Cell-Based Gene Therapies for Sickle Cell Disease

Pharmaceutical Executive Editorial Staff FDA approval of bluebird bio’s Lyfgenia and Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy marks significant milestone in the treatment of sickle cell disease. The FDA has approved a pair of landmark treatments for sickle cell disease (SCD) in patients 12 years of age and older, marking the first cell-based gene therapies for SCD. As part of today’s regulatory action, both bluebird bio’s Lyfgenia (lovotibeglogene autotemcel [lovo-cel]) and Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel [exa-cel]) gained approval for the inherited blood disorders, which affect approximately 100,000 people in the United States. “Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” Nicole Verdun, MD, director of the Office of Therapeutic Products within the ...
- Source: drugdu
- 95
- December 12, 2023
On Heels of ASH Data, Pfizer Eyes FDA Submission for New Hemophilia Drug

Pfizer already reported its experimental drug for hemophilia A and B met a pivotal study’s goals of reducing bleeding episodes. During the annual meeting of the American Society of Hematology, the company presented a full look at the results, which are expected to support regulatory submissions soon. By FRANK VINLUAN Patients with hemophilia A or B can manage the inherited bleeding disorders with regular infusions of the clotting proteins they lack. This treatment helps—until it doesn’t. One problem with these infused factor replacement therapies is patients can develop inhibitors, which are antibodies that render the clotting proteins ineffective. Experimental Pfizer drug marstacimab takes a different approach to both forms of hemophilia. Results from a pivotal study show the subcutaneously injected drug met the main goals of reducing bleeding episodes. The company also has some additional long-term data showing continuing improvement beyond the initial clinical trial evaluation period. The full Phase ...
- Source: drugdu
- 90
- December 12, 2023
FDA fast tracks Solid Biosciences’s Duchenne gene therapy

The FDA has granted fast track designation to Solid Biosciences’s gene therapy SGT-003 for the treatment of DMD. The US-based company received clearance from the FDA to start Phase I/II trial for the gene therapy on 14 November. SGT-003 uses a novel capsid AAV-SLB101 to deliver the DNA sequence encoding the shortened form of the dystrophin protein (microdystrophin). It also has R16 and R17 nNOS binding protein domains. DMD is a rare genetic condition that causes progressive muscle weakness. It affects approximately six in 100,000 individuals across Europe and North America, as per the US Muscular Dystrophy Association. The first gene therapy for treating DMD was approved earlier this year. In June, Sarepta Therapeutics’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl) received accelerated approval by the FDA for treating ambulatory patients aged 4-5 years with a confirmed mutation in the DMD gene. There has been an increased interest in developing gene therapies ...
- Source: drugdu
- 100
- December 11, 2023
Laboratory trade group, providers oppose FDA’s lab developed test proposal

Dive Brief The Food and Drug Administration received more than 19,600 comments related to its planned changes to the oversight of laboratory developed tests (LDTs). The public comment period closed on Monday. Multiple groups pushed back against plans to increase FDA oversight of LDTs. The American Clinical Laboratory Association (ACLA) is at the forefront of the resistance, submitting a 107-page document that outlines “grave concerns” and urges the FDA to withdraw the proposal. Hospitals and healthcare provider groups, like the American Hospital Association (AHA), also opposed the FDA’s proposal, arguing that the rule would limit patients’ access to tests. Dive Insight The FDA closed the comment period Monday, resisting calls to give affected groups more time to send feedback. The length of the comment period is one of many points of contention. Plans to increase regulatory oversight of LDTs, without gaining additional powers from Congress, have alarmed a spectrum of ...
- Source: drugdu
- 130
- December 9, 2023
Microbot Medical is a step closer to an FDA IDE submission

BY CHRIS NEWMARKER Microbot Medical today announced the successful completion of its pivotal pre-clinical study of its Liberty endovascular robotic surgical system. The study, performed under good laboratory practice (GLP) and essential for the company’s investigational device exemption (IDE) submission, took place under rigorous FDA guidelines. The study involved pigs. In this study, three interventional radiologists performed 96 robotic navigations using Liberty. They used the system to control various commercially available intravascular catheterization devices. They then microscopically examined and evaluated the target vessels and surrounding tissue. Microbot Medical officials think Liberty’s remote operation could enable it to democratize endovascular interventional procedures. “I am very pleased with the positive outcomes of the histopathology report and the completion of the GLP study,” said Juan Diaz Cartelle, chief medical officer of Braintree, Massachusetts–based Microbot Medical. “This gives us confidence to move forward to the next stage of human clinical study.” Microbot Medical CEO ...
- Source: drugdu
- 103
- December 9, 2023
Novartis’ Fabhalta approved by FDA as first oral monotherapy for rare blood disease PNH

Novartis’ Fabhalta (iptacopan) has been approved by the US Food and Drug Administration (FDA) as the first oral monotherapy for adults with paroxysmal nocturnal haemoglobinuria (PNH), a rare blood disease affecting approximately ten to 20 people per million worldwide. PNH patients have an acquired mutation that causes them to produce red blood cells susceptible to premature destruction by the complement system, which can cause anaemia, thrombosis, fatigue and other symptoms that can impact quality of life. The disease has a significant unmet need, Novartis reports, with a large proportion of patients on anti-C5 treatment remaining anaemic and dependent on blood transfusions. Fabhalta is a factor B inhibitor of the immune system’s complement pathway and offers an alternative to therapies that need to be delivered by infusion or injection. Victor Bultó, president US, Novartis, described the approval as an “extraordinary moment” for the PNH community. “This new, effective oral medicine may ...
- Source: drugdu
- 102
- December 8, 2023
ImmunoGen’s ADC Elahere wins FDA priority review for ovarian cancer

The US Food and Drug Administration (FDA) has granted priority review for the full approval of the antibody drug conjugate, Elahere (mirvetuximab soravtansine-gynx) and has set a Prescription Drug User Fee Act (PDUFA) action date of 5 April 2024. Elahere was a central element in the $10.1bn ImmunoGen acquisition deal announced by AbbVie last week (30 November). AbbVie expects the acquisition to complete in mid-2024. Elahere is an antibody drug conjugate (ADC) that targets folate receptor alpha (FRα). In November 2022, it was granted accelerated approval as the treatment of FRα-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer for patients who have received one to three prior systemic treatment regimens. The latest supplemental Biologics License Application (sBLA) for Elahere is supported by the data from the Phase III MIRASOL trial, which will allow for the accelerated approval to be converted to a full approval. ImmunoGen reported $105.2m in ...
- Source: drugdu
- 100
- December 7, 2023
Elli Lilly Cancer Drug Lands Its Second FDA Approval This Year

Eli Lilly cancer drug Jaypirca is closing 2023 the same way it started—with an FDA approval. The latest regulatory nod adds two additional types of blood cancers to the list of indications for the therapy. Friday’s accelerated approval for Jaypirca covers the treatment of adults with either chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The once-daily oral drug is a small molecule designed to block Bruton’s tyrosine kinase, or BTK, a cancer-driving enzyme. While other drugs already do this, Lilly’s molecule has an edge. The FDA based its decision on the results of an open-label, single-arm Phase 1/2 study in blood cancers that included more than 100 patients with CLL or SLL previously treated with at least two prior lines of therapy. Participants had received a median of five prior lines of therapy; the FDA said 77% of these patients had discontinued a BTK inhibitor after their cancer ...
- Source: drugdu
- 105
- December 6, 2023
Lilly’s Jaypirca blazes leukemia trail with FDA nod, fast confirmatory trial filing

As AbbVie, Johnson & Johnson, AstraZeneca and BeiGene are battling it out in the same BTK inhibitor market, Eli Lilly is trailblazing a new path for the blood cancer drug class. On Friday, Dec.1, the FDA granted accelerated approval to Lilly’s Jaypirca for patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least two prior lines of therapy. Jaypirca, which is itself a BTK inhibitor, is now allowed following treatment with a BTK inhibitor and a BCL-2 inhibitor. The ability to help patients who have failed on a BTK inhibitor makes Jaypirca unique. The Lilly med is a non-covalent BTK inhibitor that binds to BTK by a mechanism different from existing covalent agents, namely AbbVie/J&J’s Imbruvica, AZ’s Calquence and BeiGene’s Brukinsa. “Once patients with CLL or SLL have progressed on covalent BTK inhibitor and BCL-2 inhibitor therapies, treatments are limited and outcomes can be ...
- Source: drugdu
- 95
- December 6, 2023

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