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. Psoriasis is a widely prevalent, immune-mediated disease that affects approximately 7.5

FDA Approval 130

FDA Approval FDA Treatment Clinical Trials 130

Drug Discovery World

JUNE 23, 2023

The Food and Drug Administration (FDA) has granted accelerated approval to Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus (AAV) based gene therapy for the treatment of DMD. The FDA’s accelerated approval is based on an increase in Elevidys micro-dystrophin protein expression in skeletal muscle.

FDA Approval 243

FDA Approval Therapies FDA Protein Expression 243

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.

FDA Approval 130

FDA Approval FDA Therapies Treatment 130

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. . .

FDA Approval 130

FDA Approval FDA Therapies Disease 130

Drug Discovery World

DECEMBER 5, 2023

Jaypirca is the first FDA-approved non-covalent (reversible) BTK inhibitor. This is the marketing authorisation approval for the drug in 2023, after it was accelerated approval by the FDA for the treatment of mantle cell lymphoma (MCL) in January.

FDA Approval 130

FDA Approval FDA Therapies Treatment 130

Drug Discovery World

MARCH 26, 2024

It is approved in adults and adolescents (12 years of age and older weighing at least 40kg) who are moderate-to-severely immune compromised due to certain medical conditions or receipt of certain immunosuppressive medications or treatments and are unlikely to mount an adequate immune response to Covid-19 vaccination.

FDA Approval 130

FDA Approval FDA Clinical Trials Immune Response 130

Drug Discovery World

JUNE 12, 2023

We look forward to initiating this trial which could provide an important therapeutic option for patients suffering from this devastating orphan disease with a major unmet medical need.” ” The post FDA approves Phase III cerebral Adrenoleukodystrophy trial appeared first on Drug Discovery World (DDW).

FDA Approval 130

FDA Approval FDA Trials Clinical Trials 130

Drug Discovery World

MAY 12, 2023

Otsuka and Lundbeck’s Rexulti (brexpiprazole) has become the first pharmacological treatment to be approved in the US for agitation associated with dementia due to Alzheimer’s disease. The Food and Drug Administration (FDA) has approved the companies’ supplemental New Drug Application (sNDA).

FDA Approval 130

FDA Approval FDA Drugs Disease 130

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.

FDA Approval 100

FDA Approval FDA Virus Therapies 100

SCIENMAG: Medicine & Health

FEBRUARY 9, 2024

CLEVELAND—University Hospitals (UH) Brain Health & Memory Center is now treating patients with LEQEMBI® (lecanemab), a Food and Drug Administration-approved medication for the treatment of Alzheimer’s disease.

Hospitals 79

Hospitals FDA Approval Treatment Disease 79

Drug Discovery World

AUGUST 14, 2023

Janssen’s Akeega (niraparib and abiraterone acetate) is now available in the US for adult patients with deleterious or suspected deleterious BRCA-positive metastatic castration-resistant prostate cancer (mCRPC) following FDA approval. The European Commission (EC) granted marketing authorisation for Akeega in April 2023.

FDA Approval 130

FDA Approval FDA Treatment Disease 130

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).

FDA Approval 130

FDA Approval FDA Drugs Treatment 130

Drug Discovery World

DECEMBER 1, 2023

SpringWorks Therapeutics’ Ogsiveo (nirogacestat) has become the first drug to be approved for desmoid tumours in the US, following the go-ahead from the US Food and Drug Administration (FDA). Today’s approval will offer the first approved treatment option for patients beyond surgery and radiation.”

FDA Approval 130

FDA Approval FDA Therapies Trials 130

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).

FDA Approval 130

FDA Approval Therapies FDA Virus 130

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). CALD is a rare, progressive, neurodegenerative disease that primarily affects young boys and causes irreversible, devastating neurologic decline.

FDA Approval 130

FDA Approval FDA Therapies Hospitals 130

SCIENMAG: Medicine & Health

OCTOBER 31, 2023

. — A new drug developed by professors from the School of Pharmacy and Pharmaceutical Sciences at Binghamton University has received Food and Drug Administration (FDA) approval for the treatment of patients with Duchenne muscular dystrophy (DMD), a common genetic disease that mostly affects young boys.

FDA Approval 83

FDA Approval FDA Disease Pharmacy 83

Drug Discovery World

APRIL 6, 2023

Gohibic (vilobelimab) has been granted an Emergency Use Authorization (EUA) by the US Food and Drug Administration (FDA) for the treatment of Covid-19 in hospitalised adults. A total of 369 patients were randomly assigned to the vilobelimab treatment group (six 800mg infusions) or the placebo group.

FDA Approval 130

FDA Approval FDA Treatment Trials 130

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

FDA Approval 130

FDA Approval FDA Clinical Trials Therapies 130

PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. In 2023, two gene-based treatments became available.

FDA Approval 75

FDA Approval Treatment FDA DNA 75

Drug Discovery World

JANUARY 12, 2023

Airsupra (albuterol/budesonide) has been approved in the US for the as-needed treatment or prevention of bronchoconstriction and to reduce the risk of exacerbations in people with asthma aged 18 years and older. . Current albuterol rescue inhalers alleviate acute symptoms, but do not treat the underlying inflammation in asthma. .

FDA Approval 130

FDA Approval FDA Trials Disease 130

Drug Discovery World

JULY 14, 2022

Biotech company Actigen has moved closer to clinical trials for rare disease treatment for Hunter syndrome, following a pre-Investigational New Drug (IND) meeting with the US Food and Drug Administration (FDA). ? . Hunter syndrome . Official comments .

Clinical Trials 130

Clinical Trials Treatment Trials Disease 130

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.

FDA Approval 130

FDA Approval FDA Therapies Medical Schools 130

Drug Discovery World

NOVEMBER 14, 2023

DDW’s Megan Thomas looks at how different diseases will benefit from the success of therapeutic antibodies. What is particularly noteworthy when it comes to the mAbs being developed and approved for Covid-19 is drug repurposing, showing the scope of future treatments with mAbs. The work is continuous for this disease target.

Disease 147

Disease FDA Approval Treatment FDA 147

The Pharma Data

AUGUST 9, 2021

Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients 1 year of age and older with late-onset Pompe disease. “Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives,” said Janet Maynard, M.D.,

FDA Approval 52

FDA Approval FDA Disease Treatment 52

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.

FDA Approval 147

FDA Approval FDA Drugs Therapies 147

Drug Discovery World

OCTOBER 11, 2023

Collaborators Boehringer Ingelheim and CDR-Life have commenced a Phase I evaluation of BI 771716, their antibody fragment-based treatment developed to preserve the vision of people with geographic atrophy (GA). GA is an irreversible retinal disease that occurs in people with late-stage dry age-related macular degeneration (AMD).

Clinical Trials 130

Clinical Trials Treatment Trials FDA Approval 130

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., In the U.S.,

FDA Approval 52

FDA Approval FDA Therapies Treatment 52

Drug Discovery World

SEPTEMBER 14, 2023

6,7 “For patients with advanced UC, including FGFR-driven tumours, outcomes remain poor and treatment options are limited; therefore, there is a need for novel, targeted therapies,” said Martin Vogel, EMEA Therapeutic Area Lead Oncology, Janssen-Cilag GmbH. “We Erdafitinib for the treatment of metastatic bladder cancer. J Clin Med.

Treatment 130

Treatment Clinical Pharmacology FDA Approval Therapies 130

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.

FDA Approval 124

FDA Approval Therapies FDA Treatment 124

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.

FDA Approval 98

FDA Approval Therapies FDA Treatment 98

Drug Discovery World

MARCH 16, 2023

Overcoming Resistance to FLT3 Inhibitors in the Treatment of FLT3-Mutated AML. Around one third of patients with AML are diagnosed with FLT3-mutations, which are associated with a higher risk of relapse and poor clinical outcome. and Leung, A. International Journal of Molecular Science, 21(4): 1537. and Wang, H-G.

Treatment 130

Treatment FDA FDA Approval Drug Development 130

The Pharma Data

JUNE 28, 2023

FDA Approves Pfizer’s NGENLA™, a Long-Acting Once-Weekly Treatment for Pediatric Growth Hormone Deficiency NEW YORK & MIAMI–(BUSINESS WIRE)– Pfizer Inc. 1,2 Without treatment, children will have persistent growth attenuation, a very short height in adulthood, and puberty may be delayed.

FDA Approval 40

FDA Approval FDA Treatment Clinical Development 40

The Pharma Data

AUGUST 7, 2021

Approval is based on positive Phase 3 data demonstrating improvements in key disease burden measures and establishing its safety profile Nexviazyme specifically targets the M6P receptor, the key pathway for enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells.

FDA Approval 52

FDA Approval Disease FDA Treatment 52

Drug Target Review

MARCH 12, 2024

The study’s findings offer new insights into the biochemical language microbes use to influence distant organ systems, which could revolutionise the way researchers approach disease. For example, bile acid treatments have been effective against SARS-Cov-2 infections. Bile acids and bile activated receptors in the treatment of Covid-19.

Disease 111

Disease Biochemical Pharmacology FDA Approval Pharmacy 111

Drug Discovery World

OCTOBER 4, 2022

William Erhardt , President, Head of Development & Operations of biotech Oligomerix, tells Lu Rahman about the company’s differentiated approach to developing disease-modifying therapeutics for neurodegenerative diseases. . With its recent award of $3.35 Overcoming sector challenges .

Disease 130

Disease Small Molecule Compound Screening Clinical Development 130

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. News round-up for 18-22 March by DDW Digital Content Editor Diana Spencer.

FDA Approval 130

FDA Approval Therapies FDA Drugs 130

The Pharma Data

JANUARY 21, 2021

Today’s FDA approval of Cabenuva represents a shift in the way HIV is treated, offering people living with HIV a completely new approach to care,” said Lynn Baxter, Head of North America, ViiV Healthcare. Cabenuva reduces the treatment dosing days from 365 days to 12 days per year. Janssen is a Johnson & Johnson company.

FDA Approval 52

FDA Approval FDA Treatment Clinical Trials 52