Disease, FDA Approval, Protein Expression and Treatment

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FDA Approval

Protein Expression

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FDA approves first gene therapy for Duchenne muscular dystrophy

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

FDA Approval Therapies FDA Protein Expression

ADCs: the next generation of targeted therapies

Drug Discovery World

SEPTEMBER 25, 2024

Mylotarg was ahead of its time in many ways: its novel structure of an antibody to target diseased cells, plus a cancer-killing payload attached via a special linker, is the blueprint for the antibody-drug conjugates (ADCs) that followed.

Therapies

Therapies Pharma Companies FDA Approval FDA

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Trending Sources

Enhertu Approved in the U.S. for the Treatment of Patients With Previously Treated HER2 Positive Advanced Gastric Cancer

The Pharma Data

JANUARY 15, 2021

hereafter, Daiichi Sankyo) and AstraZeneca’s Enhertu ( fam-trastuzumab deruxtecan -nxki) has been approved in the U.S. for the treatment of adult patients with locally advanced or metastatic HER2 positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen. In the U.S.,

Treatment

Treatment Trials Disease Therapies

Roche presents new data at World Muscle Society (WMS) 2021 highlighting new advances for people living with rare neuromuscular disorders

The Pharma Data

SEPTEMBER 28, 2021

Our goal is to still lead the way in developing transformative medicines for neuromuscular diseases. We are grateful for the partnerships that are helping us to develop new therapies for people impacted by these devastating rare diseases.”. About SMA SMA may be a severe, progressive neuromuscular disease which will be fatal.

Clinical Trials

Clinical Trials Protein Expression FDA Therapies

Biopharma Money on the Move: December 2 – 8

The Pharma Data

DECEMBER 8, 2020

Some of the funds will be used for the development of therapeutics for oncology and other serious diseases through its proprietary ImmunoTAC technology platform. The ImmunoTAC platform pairs proprietary payloads that modulate key disease modifying pathways with monoclonal antibodies directed at specific disease sites.

RNA

RNA Protein Expression Therapies Disease

Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Conversations in Drug Development Trends

MAY 30, 2024

Since marketing authorization for the first breakthrough treatment in 1994, the steady increase in clinical trials reflects the community’s commitment to finding effective ALS treatments despite the numerous hurdles associated with clinical trial design, from proof-of-concept to pivotal trials. Initial approval in 2022: FDA, U.S.

Treatment

Treatment Small Molecule Clinical Trials Therapies

Five Promising Treatment Areas in Early-Phase Drug Development in 2024

Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

Drug Development

Drug Development Treatment FDA Approval Drugs

New data further reinforce Roche’s OCREVUS (ocrelizumab) as a highly effective treatment for people with multiple sclerosis

The Pharma Data

SEPTEMBER 10, 2020

75% of patients with relapsing-remitting multiple sclerosis (RRMS) and suboptimal response to prior treatment had no evidence of disease activity two years after switching to OCREVUS in open-label Phase IIIb CASTING study. 97% persistence and strong adherence to OCREVUS treatment and twice-yearly dosing schedule from real-world data.

Treatment

Treatment FDA Approval Disease Clinical Trials

Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce Extension of PDUFA Date for Pegunigalsidase Alfa for the Proposed Treatment of Fabry Disease

The Pharma Data

NOVEMBER 26, 2020

(NYSE American: PLX) (TASE: PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A.,

Disease

Disease Treatment Clinical Trials FDA

Roche expands its multiple sclerosis portfolio with investigational BTK inhibitor fenebrutinib and initiates novel clinical trials for OCREVUS (ocrelizumab)

The Pharma Data

SEPTEMBER 8, 2020

OCREVUS CHIMES study exclusively focused on disease insights and more tailored care for minority populations with MS. We remain committed to advancing the science in MS by investigating potential new medicines such as fenebrutinib, with the ultimate goal of halting progression of this disease,” said Levi Garraway, M.D.,

Clinical Trials

Clinical Trials Trials Disease FDA Approval

Roche to present new data in multiple sclerosis and neuromyelitis optica spectrum disorder at MSVirtual2020

The Pharma Data

SEPTEMBER 2, 2020

New data further reinforce OCREVUS (ocrelizumab) as a highly effective treatment option offering a favourable and consistent benefit:risk profile, with high treatment persistence and adherence. Initiation of Phase IIIb OCREVUS higher dose clinical trial programme and Phase IV study evaluating OCREVUS in minority populations.

Clinical Trials

Clinical Trials Disease Treatment Therapies

2021 AAN highlight impact and breadth of expanding neuroscience portfolio

The Pharma Data

APRIL 8, 2021

OCREVUS data show its consistent benefit on slowing disease progression in relapsing multiple sclerosis (RMS) and primary progressive MS (PPMS). Data for ENSPRYNG in neuromyelitis optica spectrum disorder (NMOSD) reinforce safety and efficacy, including in patients with concomitant autoimmune diseases (CAIDs). Following U.S.

Clinical Trials

Clinical Trials Trials Disease Nurses

BioSpace Global Roundup: Cevec Licenses AAV Program to Biogen

The Pharma Data

JANUARY 13, 2021

Food and Drug Administration gave it the green light to initiate a Phase III study of tesamorelin for the treatment of adults with Nonalcoholic Steatohepatitis (NASH) with liver fibrosis. Vivity received FDA approval in February 2020. provided an update on its ongoing NASH program. This week, the company said the U.S.

Licensing

Licensing Licensing Clinical Trials Vaccine

Transgene Announces Detailed Results From Clinical Study of TG4001 in Combination With Avelumab in Advanced HPV-positive Cancers

The Pharma Data

OCTOBER 26, 2020

The treatment induced HPV-specific T-cell responses and was associated with increased levels of immune cell infiltration in the tumors and expression of genes associated with activation of the immune system. PBMC and tissue samples were collected longitudinally prior to and during the treatment period.

Vaccine

Vaccine Virus Trials Treatment

Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce Final Results of BRIDGE Phase III Open-Label, Switch-Over Clinical Trial Evaluating Pegunigalsidase Alfa for the Treatment of Fabry Disease

The Pharma Data

DECEMBER 29, 2020

(NYSE American:PLX) (TASE:PLX), a biopharmaceutical company focused on the development, production and commercialization of recombinant therapeutic proteins produced by its proprietary ProCellEx ® plant cell-based protein expression system, and Chiesi Global Rare Diseases , a business unit of Chiesi Farmaceutici S.p.A.,

Clinical Trials

Clinical Trials Disease Treatment Trials

Drug Discovery Digest

FDA approves first gene therapy for Duchenne muscular dystrophy

ADCs: the next generation of targeted therapies

Trending Sources

Enhertu Approved in the U.S. for the Treatment of Patients With Previously Treated HER2 Positive Advanced Gastric Cancer

Roche presents new data at World Muscle Society (WMS) 2021 highlighting new advances for people living with rare neuromuscular disorders

Biopharma Money on the Move: December 2 – 8

Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Five Promising Treatment Areas in Early-Phase Drug Development in 2024

New data further reinforce Roche’s OCREVUS (ocrelizumab) as a highly effective treatment for people with multiple sclerosis

Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce Extension of PDUFA Date for Pegunigalsidase Alfa for the Proposed Treatment of Fabry Disease

Roche expands its multiple sclerosis portfolio with investigational BTK inhibitor fenebrutinib and initiates novel clinical trials for OCREVUS (ocrelizumab)

Roche to present new data in multiple sclerosis and neuromyelitis optica spectrum disorder at MSVirtual2020

2021 AAN highlight impact and breadth of expanding neuroscience portfolio

BioSpace Global Roundup: Cevec Licenses AAV Program to Biogen

Transgene Announces Detailed Results From Clinical Study of TG4001 in Combination With Avelumab in Advanced HPV-positive Cancers

Protalix BioTherapeutics and Chiesi Global Rare Diseases Announce Final Results of BRIDGE Phase III Open-Label, Switch-Over Clinical Trial Evaluating Pegunigalsidase Alfa for the Treatment of Fabry Disease

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