Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

FDA Approval 130

FDA Approval Therapies FDA Disease 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). The post FDA approves gene therapy for rare neurological disorder appeared first on Drug Discovery World (DDW).

FDA Approval 130

FDA Approval FDA Therapies Hospitals 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

AUGUST 5, 2024

Adaptimmune Therapeutics’ Tecelra (afamitresgene autoleucel) has become the first engineered cell therapy for a solid tumour cancer approved in the US. It is also the first new therapy option in more than a decade for synovial sarcoma, which is a rare, soft tissue cancer that most commonly impacts young adults.

Engineering 262

Engineering Therapies FDA Approval FDA 262

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.

FDA Approval 130

FDA Approval FDA Therapies Medical Schools 130

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). The FDA approval of Izervay is great news for the retina community and our patients suffering from GA.”

FDA Approval 130

FDA Approval FDA Clinical Trials Clinical Research 130

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.

FDA Approval 130

FDA Approval FDA Therapies Treatment 130

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

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

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

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. The post This week in drug discovery (19-23 August) appeared first on Drug Discovery World (DDW).

FDA Approval 147

FDA Approval FDA Drugs Therapies 147

Drug Discovery World

AUGUST 21, 2023

Ipsen’s Sohonos (palovarotene) has become the first drug approved for patients with fibrodysplasia ossificans progressiva (FOP) in the US, following the granting of a marketing authorisation by the Food and Drug Administration (FDA). The EU’s regulator chose not to recommend the drug in January 2023.

FDA Approval 130

FDA Approval FDA Treatment Regulations 130

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.

Therapies 147

Therapies Clinical Trials Trials FDA Approval 147

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.

FDA Approval 130

FDA Approval FDA Clinical Trials Therapies 130

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.

FDA Approval 130

FDA Approval FDA Clinical Trials Trials 130

Drug Discovery World

APRIL 23, 2024

Michael Wyand, PhD, Chief Executive Officer/Director, Oxeia Biopharmaceuticals explains why there’s a need for new drugs to treat traumatic brain injuries. While those therapies provide some relief to concussed patients, 20% or more continue to have persistent symptoms for weeks to months to years after injury.

Therapies 147

Therapies Drugs Laboratories Medical Schools 147

Drug Discovery World

JUNE 12, 2023

The US Food and Drug Administration (FDA) has approved Minoryx Therapeutics’ Phase III clinical trial (CALYX) of leriglitazone, to treat adult male X-linked Adrenoleukodystrophy (X-ALD) patients with cerebral Adrenoleukodystrophy (cALD).

FDA Approval 130

FDA Approval FDA Trials Clinical Trials 130

Drug Discovery World

SEPTEMBER 12, 2024

Childhood cancers often have a different biology than those in adults, yet targeted treatment options are mostly based on adult therapies. Only two drugs were approved exclusively for children and young people with cancer by the European Medicines Agency (EMA) and five by the US Food and Drug Administration (FDA) from 2007 to 2022.

Research 147

Research Small Molecule FDA Approval Drugs 147

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.

Therapies 162

Therapies Clinical Trials FDA Approval Virus 162

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 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).

FDA Approval 130

FDA Approval Therapies FDA Drugs 130

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).

FDA Approval 130

FDA Approval FDA Treatment Clinical Trials 130

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%

Therapies 130

Therapies FDA Approval FDA Clinical Trials 130

Drug Discovery World

AUGUST 9, 2024

The news highlights this week all centre around significant US Food and Drug Administration (FDA) regulatory decisions, including new approvals for drugs for solid tumours, hepatitis B and glioma. The post This week in drug discovery (5-9 August) appeared first on Drug Discovery World (DDW).

FDA 130

FDA FDA Approval Engineering Drugs 130

Drug Discovery World

FEBRUARY 23, 2024

In a breakthrough for advanced therapies, this week saw the FDA approve the first ever cell therapy for solid tumour cancers, but there were other significant developments in the cell and gene therapy space. The post This week in drug discovery (19-23 February) appeared first on Drug Discovery World (DDW).

FDA Approval 130

FDA Approval Therapies Licensing Licensing 130

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). Development of the drug was funded by $2.2 This is a victory for the entire ALS community, which came together to advocate for early approval of AMX0035.

FDA Approval 130

FDA Approval FDA Treatment Clinical Trials 130

Drug Discovery World

OCTOBER 12, 2023

In the In Conversation With series, a part of the free DDW podcast, DDW speaks with members of the drug discovery industry about their work and how it helps turn science into business. In this episode, Megan Thomas is in conversation with Dr Jude Samulski, Co-founder and Chief Scientific Officer at AskBio.

Therapies 130

Therapies FDA Approval Clinical Trials Virus 130

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.

Therapies 147

Therapies FDA Approval FDA Government 147

Drug Discovery World

OCTOBER 12, 2023

In the In Conversation With series, a part of the free DDW podcast, DDW speaks with members of the drug discovery industry about their work and how it helps turn science into business. In this episode, Megan Thomas is in conversation with Dr Jude Samulski, Co-founder and Chief Scientific Officer at AskBio.

Therapies 130

Therapies FDA Approval Clinical Trials Virus 130

Drug Discovery World

DECEMBER 8, 2023

Andrew Busey , Co-Founder, Form Bio discusses why vertical AI is set to reshape the cell and gene therapy sector. The cell and gene therapy industry is no exception to this digital transformation. General AI tools such as ChatGPT have acted as a catalyst, igniting a wave of AI innovation akin to the early days of the internet era.

Therapies 162

Therapies Clinical Trials Small Molecule Production 162

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). FRIDAY, June 23, 2023 -- The U.S. The groundbreaking treatment will not be cheap:

FDA Approval 111

FDA Approval Therapies FDA Treatment 111

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). FRIDAY, June 23, 2023 -- The U.S. The groundbreaking treatment will not be cheap:

FDA Approval 98

FDA Approval Therapies FDA Treatment 98

Drug Discovery World

MARCH 18, 2024

The US Food and Drug Administration (FDA) has granted orphan drug and paediatric exclusivity to Cresemba (isavuconazonium sulfate) for the treatment of invasive aspergillosis (IA) and invasive mucormycosis (IM) in paediatric patients. Paediatric exclusivity extends that period by another six months. .”

FDA 130

FDA FDA Approval Drugs Treatment 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.,

FDA Approval 52

FDA Approval FDA Therapies Treatment 52

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). The post First therapy for very rare blood clotting disorder gets US greenlight appeared first on Drug Discovery World (DDW).

Therapies 130

Therapies FDA Approval Protein Production FDA 130