Drug Discovery World

AUGUST 7, 2024

The US Food and Drug Administration (FDA) has cleared GigaGen’s application to initiate a Phase I trial of its polyclonal drug for the treatment of hepatitis B virus (HBV) infection, GIGA-2339. With over 1,000 different HBV-targeted antibodies in the mixture, GIGA-2339 is unlike any therapy currently in development.

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Drug Discovery World

AUGUST 14, 2024

The US Food and Drug Administration (FDA) has approved a Phase II clinical trial for Biosyngen’s BRG01, an EBV-specific CAR-T cell therapy. This marks the first cell therapy to enter Phase lI trials in both the US and China for the treatment of relapsed/metastatic EBV-positive nasopharyngeal carcinoma.

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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.

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Drug Discovery World

JUNE 25, 2024

The US Food and Drug Administration (FDA) has expanded its approval for Elevidys (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least four years of age. The FDA granted accelerated approval for non-ambulatory patients.

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Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. 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.

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Drug Discovery World

MAY 6, 2024

Food and Drug Administration (FDA). The company’s lead therapeutic peptide candidate is currently in a Phase III clinical trial for the treatment of hypoparathyroidism. Amolyt Pharma has announced that its lead therapeutic peptide candidate, eneboparatide, has been granted fast track designation by the U.S.

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Drug Discovery World

FEBRUARY 19, 2024

The US Food and Drug Administration has approved Amtagvi (lifileucel), the first cellular therapy indicated for the treatment of metastatic, unresectable melanoma. Amtagvi is the first FDA-approved tumour-derived T cell immunotherapy. Iovance is also conducting TILVANCE-301, a Phase III trial to confirm clinical benefit.

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Drug Discovery World

JULY 31, 2024

The US Food and Drug Administration (FDA) has granted Fast Track designation to Tonix Pharmaceuticals’ Tonmya (cyclobenzaprine HCl sublingual tablets) for the management of fibromyalgia. It plans to submit the new drug application (NDA) to the FDA in the second half of 2024.

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Drug Discovery World

JANUARY 5, 2024

Kyverna Therapeutics has revealed plans to progress to Phase II trials of KYV-101 for multiple sclerosis (MS) following a green light from the US Food and Drug Administration (FDA). CAR T-cell therapy involves modifying a patient’s T cells to recognise and remove B cells in the patient’s body.

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Drug Discovery World

OCTOBER 4, 2023

The US Food and Drug Administration (FDA) has accepted Rocket Pharmaceuticals’ Biologics License Application (BLA) and granted Priority Review for RP-L201 (marnetegragene autotemcel). The post FDA acceptance advances investigational gene therapy appeared first on Drug Discovery World (DDW).

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Drug Discovery World

OCTOBER 3, 2023

The US Food and Drug Administration (FDA) has announced a pilot programme with the aim of accelerating the development of novel drug and biological products for rare diseases. The FDA will be accepting applications to the START programme between 2 January 2024 and 1 March 2024.

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Drug Discovery World

JULY 12, 2024

The US Food and Drug Administration (FDA) has granted Fast Track Designation for Asklepios BioPharmaceutical’s (AskBio) AB-1005 which is being developed for moderate Parkinson’s disease. AB-1005 is an investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy.

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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). The post FDA approves first therapy for rare non-cancerous tumours appeared first on Drug Discovery World (DDW).

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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

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

AUGUST 20, 2024

The US Food and Drug Administration (FDA) has approved axatilimab-csfr (Niktimvo), a colony stimulating factor-1 receptor-blocking antibody, for the treatment of chronic graft-versus-host disease (cGVHD). ORR was 75% in the 79 patients treated with the recommended dosage. The median time to first response was 1.5

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Drug Discovery World

MARCH 6, 2024

UK-based biotech SynaptixBio has secured a second Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA). This second ODD allows research and development of a therapy for another form of the disease, Isolated Hypomyelination.

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Drug Discovery World

JULY 17, 2024

The US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to Kyverna Therapeutic’s CAR-T candidate KYV-101 for refractory stiff-person syndrome (SPS). KYV-101 is an autologous, fully human CD19 CAR-T cell product candidate for use in B cell-driven autoimmune diseases.

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Drug Discovery World

DECEMBER 2, 2022

The US Food and Drug Administration (FDA) has accepted Sarepta Therapeutics’ Biologics License Application (BLA) seeking accelerated approval of SRP-9001 (delandistrogene moxeparvovec) for the treatment of ambulant individuals with Duchenne muscular dystrophy. .

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Drug Discovery World

DECEMBER 20, 2023

The US Food and Drug Administration (FDA) has approved antibody-drug conjugate (ADC) enfortumab vedotin-ejfv (Padcev) in combination with pembrolizumab (Keytruda) for patients with locally advanced or metastatic urothelial cancer (la/mUC). In the Phase III EV-302 trial, median overall survival (OS) was 31.5 months compared to 6.3

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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

JUNE 15, 2023

A Phase I clinical trial testing a CAR-T cell therapy treatment in children with acute myeloid leukaemia (AML) has been paused due to a patient’s death. The PLAT-08 clinical trial is a Phase I study of 2seventy bio’s cell therapy SC-DARIC33 in patients with AML.

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Drug Discovery World

OCTOBER 9, 2023

Positive Phase I trial data indicates that Reqorsa (quaratusugene ozeplasmid) has shown early efficacy for the treatment of non-small cell lung cancer (NSCLC). Developed by gene therapy company Genprex, Reqorsa is a non-viral gene therapy that leads to expression of the TUSC2 tumour suppressor gene in cancers.

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Drug Discovery World

OCTOBER 27, 2023

The US Food and Drug Administration (FDA) has granted clearance for Endogena’s Investigational New Drug (IND) application for EA 2351, a potential treatment for geographic atrophy (GA). The post FDA gives green light for AMD treatment trials appeared first on Drug Discovery World (DDW).

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Drug Discovery World

FEBRUARY 21, 2024

DDW Editor Reece Armstrong speaks to Dr Paul Song , MD, NKGen and Dr Vince DeOrchis , clinician, to find out more about the investigational natural killer therapy SNK01 and the benefit of using a patient’s own cells to treat Alzheimer’s. PS : Most, if not all, current therapies are focused on MCI. RA: How does SNK01 work?

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Drug Discovery World

FEBRUARY 2, 2023

Orserdu was approved under the FDA’s Priority Review and Fast Track designation based on the results of the registrational Phase III trial EMERALD, that demonstrated statistically significant progression-free survival (PFS) with elacestrant vs. SOC endocrine monotherapy.

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Drug Discovery World

NOVEMBER 16, 2023

The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Trial results for Casgevy The authorisation is based on positive clinical trial data.

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Drug Discovery World

JULY 8, 2024

Biopharmaceutical company Syncromune has had its lead-candidate for the treatment of metastatic castrate-resistant prostate cancer (mCRPC) granted Fast Track designation by the FDA. We look forward to initiating trials at multiple US sites later this year to expand our efforts to develop the SYNC-T SV-102 Therapy.”

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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

MARCH 1, 2024

The US Food and Drug Administration (FDA) has announced its final decision to withdraw approval of Pepaxto (melphalan flufenamide), despite an appeal by manufacturer Oncopeptides. Oncopeptides was required to conduct the OCEAN trial as a post-approval requirement under the accelerated approval programme.

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Drug Discovery World

NOVEMBER 30, 2023

The Food and Drug Administration (FDA) said it will investigate the safety of BCMA- or CD19-directed autologous CAR-T cell immunotherapies, following reports of T cell malignancies, including chimeric antigen receptor CAR-positive lymphoma, in these patients.

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Drug Discovery World

JULY 24, 2024

CHOP identified a candidate antisense oligonucleotide (ASO) as a potential therapy for H-ABC. This extension to the companies’ collaboration agreement will see Evotec significantly broaden the pipeline with additional ASOs as candidate therapies. They are instrumental in supporting us as we prepare for those clinical trials.”

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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

JUNE 26, 2024

Biotechnology company EG 427 has revealed plans to start a Phase Ib/IIa clinical study in neurogenic detrusor overactivity (NDO), following Investigational New Drug (IND) clearance from the US Food and Drug Administration (FDA). It is a step forward in bringing the potential benefits of gene therapy to large disease populations in neurology.

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Drug Discovery World

MARCH 8, 2024

The US Food and Drug Administration (FDA) has revealed plans to convene a meeting of the Peripheral and Central Nervous System Drugs Advisory Committee (PCNS) to discuss the Phase III TRAILBLAZER-ALZ 2 trial. The trial evaluated the efficacy and safety of Eli Lilly’s donanemab in early symptomatic Alzheimer’s disease.

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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

FEBRUARY 29, 2024

The stock market value of Minerva Biosciences has dropped nearly 60%, following the US Food and Drug administration (FDA) decision not to approve roluperidone for symptoms of schizophrenia. The company received a ‘Complete Response Letter’ from the FDA highlighting the clinical deficiencies in its new drug application (NDA).

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