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.

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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).

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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). As a condition of the SKYSONA Accelerated Approval, the manufacturer will provide confirmatory long-term clinical data to the FDA.

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Drug Discovery World

AUGUST 11, 2023

The new complement C5 inhibitor is the only approved GA treatment with a statistically significant reduction in the rate of GA progression at the 12-month primary endpoint across two Phase III clinical trials. The FDA approval of Izervay is great news for the retina community and our patients suffering from GA.”

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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

FEBRUARY 2, 2023

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

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. Setting a standard For CGTs to find success in the journey from clinic to bedside, everything from clinical trial design through to quality control requires standardisation.

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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.

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Drug Discovery World

JULY 25, 2023

The efficacy of quizartinib with chemotherapy was evaluated in the QuANTUM-First trial of 539 patients with newly diagnosed FLT3-ITD positive AML. FLT3-ITD status was determined prospectively with a clinical trial assay and verified retrospectively with the companion diagnostic LeukoStrat CDx FLT3 Mutation Assay.

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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

MARCH 22, 2024

Jean-Paul Clozel, MD and Chief Executive Officer of Idorsia, commented: “Today, there are millions of Americans whose blood pressure is not well-controlled despite existing therapies. ” The post FDA approval represents ‘transformational progress’ in hypertension appeared first on Drug Discovery World (DDW).

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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

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

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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Drug Discovery World

OCTOBER 4, 2022

The approval is the result of a long advocacy campaign to convince the FDA to approve the treatment prior to completion of an ongoing Phase III trial. . “We In June 2016, the Association provided Amylyx, the producer of AMX0035, with a $750,000 grant for a clinical trial pilot.

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Drug Discovery World

OCTOBER 12, 2023

Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field. His work remains a key component of every FDA-approved AAV therapeutic and will remain central to future advances. The post The evolution and future of gene therapy appeared first on Drug Discovery World (DDW).

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Drug Discovery World

OCTOBER 12, 2023

Dr Samulski’s discoveries in AAV played a profound role in shaping the evolution of the gene therapy field. His work remains a key component of every FDA-approved AAV therapeutic and will remain central to future advances. The post Podcast: The evolution and future of gene therapy appeared first on Drug Discovery World (DDW).

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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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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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BioPharma Drive: Drug Pricing

JUNE 4, 2024

A group of independent experts wasn't convinced by clinical trial data from company Lykos Therapeutics, which is seeking FDA approval of MDMA-assisted treatment for post-traumatic stress disorder.

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

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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).

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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. In addition, small patient populations make clinical trials difficult and reduce incentives for investment. In contrast, 14 new cancer medicines for adults received FDA approval in 2023 alone.

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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. For those patients who had received prior therapy, the ORR by IRR was 46%, including 10% CRs and 36% PRs at primary analysis.

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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.

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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. The New Drug Duvyzat (givinostat), a type of drug called an HDAC inhibitor, has been in clinical trials to treat cancers and other disorders of the blood, Crohn’s disease, and a form of juvenile arthritis.

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Drug Discovery World

APRIL 3, 2023

This year, the team will launch a combined Phase I and IIa trial of R805/CX-011 for the treatment of osteoarthritis in patients in collaboration with the start-up Carthronix. They think the drug has the potential to delay or even reduce the need for joint replacement surgery, the only existing FDA-approved therapy for osteoarthritis.

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Drug Discovery World

MARCH 29, 2024

The top stories: Novo Nordisk acquires cardiovascular therapies developer Novo Nordisk has acquired Cardior Pharmaeuticals for up to €1.025 billion, including an upfront payment and additional payments if certain milestones are achieved. News round-up for 25-29 March by DDW Digital Content Editor Diana Spencer.

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Drug Discovery World

JUNE 16, 2023

Regulatory approvals and industry guidelines are key to the continued development and discovery of drugs, which is a common theme throughout this week’s news highlights, including an FDA approval for Pfizer, a NICE recommendation for AstraZeneca UK, and an update to the HER2 Breast Cancer Testing Guidelines.

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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 .

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Drug Discovery World

MAY 1, 2024

Many of the best-selling drugs in 2023 were monoclonal antibody (mAB) therapies, including Keytruda (pembrolizumab), Humira (adalimumab), and Dupixent (dupilumab) 1. Hammond says: “Many of the most popular drugs in 2023 are mAb-based therapies, which we predict will continue in 2024.

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The Pharma Data

AUGUST 24, 2020

The FDA has approved a request from American Gene Technologies to begin a clinical study into its HIV gene therapy. In a paper published in Molecular Therapy: Methods & Clinical Development , the team discussed why they choose this type of gene therapy to treat HIV. Air Force photo by Kemberly Groue.

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The Pharma Data

DECEMBER 21, 2020

FDA Approves First Oral Hormone Therapy for Advanced Prostate Cancer. 21, 2020 — Orgovyx (relugolix) is now approved to treat advanced prostate cancer and is the first oral hormone therapy approved for this indication, the U.S. Professional. MONDAY, Dec. Food and Drug Administration announced Friday.

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Drug Discovery World

JUNE 6, 2024

The combination therapy was also shown to reduce the risk of distant metastasis or death by 62% compared to Keytruda alone in these patients. The global Phase III clinical trial is being led by University College London Hospitals NHS Foundation Trust in the UK and will include around 1,100 people.

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Drug Discovery World

MARCH 1, 2024

Pepaxto associated with increased risk of death The FDA is alerting patients and health care professionals that a clinical trial (OCEAN, Study OP-103) evaluating Pepaxto with dexamethasone showed an increased risk of death. The FDA has now suspended enrolment in all ongoing Pepaxto clinical trials.

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Drug Discovery World

JULY 7, 2023

It’s been a fascinating week in drug discovery, as AstraZeneca announced that its Phase III trial of datopotamab deruxtecan did not meet its second primary endpoint and saw an 8% drop in its stock market value as a result.

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Drug Discovery World

DECEMBER 21, 2023

In a record year for gene therapy approvals, it isn’t surprising that cell & gene therapy (CGT) has been a hot topic. Regulatory issues and diversity in clinical trials were also important in 2023, as well as the role that artificial intelligence tools like ChatGPT might play.

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