Drug Discovery World

DECEMBER 1, 2023

Although surgical removal has historically been the treatment of choice, there is a high risk that the tumour will return or that other health challenges will occur after removal. Today’s approval will offer the first approved treatment option for patients beyond surgery and radiation.”

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Drug Discovery World

SEPTEMBER 20, 2022

SKYSONA has become the first FDA approved therapy to slow the progression of neurologic dysfunction in boys with cerebral adrenoleukodystrophy (CALD). Nearly half of patients who do not receive treatment die within five years of symptom onset.

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Drug Discovery World

DECEMBER 20, 2023

The overall survival benefit seen in the EV-302 trial demonstrates the potential for Padcev in combination with pembrolizumab to impact first-line treatment of patients with locally advanced or metastatic urothelial carcinoma. ” Enfortumab vedotin-ejfv was co-developed by Pfizer and Astellas.

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Drug Discovery World

AUGUST 21, 2023

The FDA approval is based on data from the Phase III MOVE trial, which demonstrated that palovarotene effectively reduced annualised heterotopic ossification (HO) volume compared with no treatment beyond standard of care (54% reduction with weighted linear mixed effect model).

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Drug Discovery World

MAY 29, 2023

The US Food and Drug Administration has approved Krystal Biotech’s Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of dystrophic epidermolysis bullosa (DEB). The post FDA approves first gene therapy for rare skin disorder appeared first on Drug Discovery World (DDW).

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Drug Discovery World

SEPTEMBER 2, 2024

The European Commission (EC) has approved Braftovi (encorafenib) in combination with Mektovi (binimetinib) for the treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with a BRAFV600E mutation.

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Drug Discovery World

AUGUST 11, 2023

The US Food and Drug Administration (FDA) has approved Izervay (avacincaptad pegol intravitreal solution) for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Slowing of disease progression was observed as early as six months with up to a 35% reduction in the first year of treatment.

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Drug Discovery World

FEBRUARY 2, 2023

The first and only treatment specifically indicated for patients with ESR1 mutations in ER+, HER2- advanced or metastatic breast cancer has been approved in the US. The approval of elacestrant is welcomed as it offers a novel option for patients with ER+, HER2- metastatic breast cancer.

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Drug Discovery World

DECEMBER 5, 2023

It has been granted approval by the Food and Drug Administration (FDA) to treat patients who have received at least two lines of therapy, including a Bruton’s tyrosine kinase (BTK) inhibitor and a BCL-2 inhibitor. Jaypirca is the first FDA-approved non-covalent (reversible) BTK inhibitor.

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Drug Discovery World

SEPTEMBER 13, 2022

The US Food and Drug Administration (FDA) has approved Bristol Myers Squibb’s allosteric tyrosine kinase 2 inhibitor Sotyktu (deucravacitinib) for the treatment of adults with moderate-to-severe plaque psoriasis. The post FDA approves new first-line treatment for plaque psoriasis appeared first on Drug Discovery World (DDW).

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Drug Discovery World

MARCH 22, 2024

The US Food and Drug Administration (FDA) has approved Tryvio (aprocitentan) for the treatment of hypertension in combination with other antihypertensive drugs, to lower blood pressure in adult patients who are not adequately controlled on other drugs.

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Drug Discovery World

OCTOBER 4, 2022

The US Food and Drug Administration (FDA) has granted approval to AMX0035, a new treatment for people living with amyotrophic lateral sclerosis (ALS). million from the ALS Association, raised by the 2014 social media craze the ‘Ice Bucket Challenge’ Another 40 potential new treatments are being investigated. .

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Drug Discovery World

NOVEMBER 21, 2022

The US Food and Drug Administration (FDA) has approved the Biologics License Application (BLA) for anti-CD3-directed antibody TZIELD (teplizumab-mzwv), the first immunomodulatory treatment for type 1 diabetes (T1D). . The company also offers financial assistance options to eligible patients for out-of-pocket costs. . .

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Drug Discovery World

SEPTEMBER 2, 2024

DDW’s Megan Thomas discusses the future of the cell and gene therapy (CGT) sector with industry experts, who predict what to expect in 2024 and beyond. This paired with next generation sequencing technologies helping to identify potential gene therapy candidates for more effective and targeted interventions will be powerful.

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Drug Discovery World

JULY 20, 2023

The US Food and Drug Administration (FDA) has granted marketing authorisations to several new therapeutics over the last month that will have a significant impact on areas of unmet need and the evolution of future treatments.

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Drug Discovery World

JULY 25, 2023

The US Food and Drug Administration (FDA) has approved Daiichi Sankyo’s quizartinib (Vanflyta) for the treatment of adult patients with newly diagnosed acute myeloid leukaemia (AML) that is FLT3 internal tandem duplication (ITD)-positive.

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Drug Discovery World

MAY 15, 2023

The US Food and Drug Administration (FDA) has given the go-ahead to CytoAgents to initiate a US Phase Ib/IIa clinical trial investigating CTO1681 for cytokine release syndrome (CRS) in lymphoma patients receiving CAR-T cell therapy. The company has also announced the initial closing of its second equity round of financing. “We

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Drug Discovery World

JUNE 28, 2023

Verona Pharma has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for approval of ensifentrine for the maintenance treatment of patients with chronic obstructive pulmonary disease (COPD).

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Drug Discovery World

JUNE 12, 2023

The NEXUS interim analysis showed that after 24 weeks of treatment all patients were clinically stable and, radiologically, demonstrated disease arrest or lesion growth stabilisation. Radiological changes were similar to those attained with Hematopoietic Stem Cell Transplant (HSCT) or ex-vivo gene therapy.

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PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Results from the study that led to the FDA approval appeared in The Lancet Neurology in April 2024 with commentary. Researchers have been working on developing gene therapy for DMD for decades.

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Drugs.com

DECEMBER 8, 2023

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology.Casgevy, developed by Vertex. FRIDAY, Dec. 8, 2023 -- The U.S.

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Drugs.com

DECEMBER 8, 2023

Food and Drug Administration on Friday approved two milestone gene therapies for sickle cell disease, including the first treatment ever approved that uses gene-editing technology. FRIDAY, Dec. 8, 2023 -- The U.S. Casgevy, developed by Vertex.

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Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies. Adeno-associated virus (AAV) vector-based gene therapies hold exceptional promise across a range of disease areas.

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Drug Discovery World

AUGUST 23, 2024

It’s been a busy week for medical regulatory agencies, with a number of key decisions made on therapies for graft-versus-host-disease, neuroendocrine carcinoma, haemophilia B, lymphoma and non-small cell lung cancer. News round-up for 19-23 August by DDW Senior Digital Content Editor Diana Spencer.

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Drug Discovery World

FEBRUARY 14, 2024

AlphaMedix is a targeted alpha therapy currently in Phase II clinical development, which consists of an SSTR-targeting peptide complex radiolabeled with lead-212 (212Pb) that serves as an in vivo generator of alpha particles. In the Phase I study, treatment was well-tolerated, with a response rate of 62.5%

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BioPharma Drive: Drug Pricing

FEBRUARY 21, 2024

One expert views Amtagvi’s approval as a catalyst for further investment in TIL therapies, akin to how Kymriah’s 2017 clearance buoyed CAR-T treatment.

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Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

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Drugs.com

FEBRUARY 19, 2024

Food and Drug Administration has approved a novel treatment for advanced melanoma, the most deadly form of skin cancer. becomes the first cellular therapy approved to. MONDAY, Feb. 19, 2024 -- The U.S. Amtagvi, made by Iovance Biotherapeutics Inc.,

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Drugs.com

JUNE 23, 2023

Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD). The groundbreaking treatment will not be cheap: FRIDAY, June 23, 2023 -- The U.S.

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Drugs.com

FEBRUARY 19, 2024

Food and Drug Administration has approved a novel treatment for advanced melanoma, the most deadly form of skin cancer.Amtagvi, made by Iovance Biotherapeutics Inc., becomes the first cellular therapy approved to. MONDAY, Feb. 19, 2024 -- The U.S.

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Drug Discovery World

FEBRUARY 15, 2024

DDW Editor Reece Armstrong speaks to Tracy M Woody , President and Chief Executive Officer of Cessation Therapeutics, about the company’s goal to develop an antibody therapy to prevent fentanyl overdoses. As it stands, there are no FDA-approved products designed to prevent a fentanyl overdose.

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Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

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Drugs.com

JUNE 23, 2023

Food and Drug Administration on Thursday approved the drug Elevidys, the first gene therapy for the treatment of children with Duchenne muscular dystrophy (DMD). The groundbreaking treatment will not be cheap: FRIDAY, June 23, 2023 -- The U.S.

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Drug Discovery World

NOVEMBER 14, 2023

Takeda’s Adzynma has become the first recombinant protein product approved in the US for prophylactic or on-demand enzyme replacement therapy (ERT) for congenital thrombotic thrombocytopenic purpura (cTTP). Without treatment, cTTP is ultimately fatal.

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The Pharma Data

JUNE 2, 2023

Food and Drug Administration (FDA) for the treatment of adult patients with deleterious or suspected deleterious BRCA -mutated ( BRCA m) metastatic castration-resistant prostate cancer (mCRPC). Patients should be selected for therapy based on an FDA-approved companion diagnostic for LYNPARZA. In the U.S.,

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Drug Discovery World

APRIL 23, 2024

Given the increased awareness of concussion and the associated long-term sequela from concussive injury it is imperative that we accelerate drug development efforts to find an effective therapy. Tremendous strides have been made with other diseases, yet there are still no FDA-approved drug therapies for TBI.

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Drug Discovery World

MARCH 22, 2024

This has been an interesting week for cell and gene therapies, with two landmark FDA approvals, two significant fundraising efforts and potentially ground-breaking study results in glioblastoma. The post This week in drug discovery (18-22 March) appeared first on Drug Discovery World (DDW).

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