FDA Approval, Protein Expression and Therapies

FDA Approval

Protein Expression

Therapies

FDA approves first gene therapy for Duchenne muscular dystrophy

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.

FDA Approval

FDA Approval Therapies FDA Protein Expression

mRNA Cancer Vaccines and Therapies: An Overview

Advarra

JANUARY 11, 2024

The applications of mRNA-based therapies in cancer research represent one of the next groundbreaking steps toward improved cancer treatments. Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials.

Vaccine

Vaccine Therapies Clinical Trials Small Molecule

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Trending Sources

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

The presentations included additional results from the RAINBOWFISH study, evaluating the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic spinal muscular atrophy (SMA) from birth to 6 weeks aged and data supporting the continued clinical investigation of gene therapy, SRP-9001, in Duchenne dystrophy (DMD).

Clinical Trials

Clinical Trials Protein Expression FDA Therapies

Biopharma Money on the Move: December 2 – 8

The Pharma Data

DECEMBER 8, 2020

Since partnering with Eli Lilly to produce the first monoclonal antibody therapy approved for mild-to-moderate COVID-19 patients, antibody discovery company AbCellera is going big on the Nasdaq. BioAge is on the cusp of taking pilot therapies BGE-117 and BGE-175 into clinical trials, targeting the first half of 2021.

RNA

RNA Protein Expression Therapies Disease

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

Enhertu is also approved in Japan for HER2 positive unresectable advanced or recurrent gastric cancer that has progressed after chemotherapy based on the DESTINY-Gastric01 trial. Enhertu previously received Priority Review and Breakthrough Therapy Designation (BTD) in the U.S. Targeted Therapies for Stomach Cancer. N Engl J Med.

Treatment

Treatment Trials Disease Therapies

Present Treatments & Hopeful Future Directions for ALS Pharmacotherapies

Conversations in Drug Development Trends

MAY 30, 2024

A condition of approval is the completion of a Phase 3 study designed to evaluate pheno-conversion in SOD1 mutation carriers (ATLAS study) to confirm clinical benefit. Moreover, exploring the repurposing of existing drugs offers additional pathways to potentially effective treatments through adopting a horizontal thinking-based approach.

Treatment

Treatment Small Molecule Clinical Trials Therapies

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

Alta Sciences

APRIL 17, 2024

Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.

Drug Development

Drug Development Treatment FDA Approval Therapies

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

The Pharma Data

SEPTEMBER 10, 2020

Basel, 11 September 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new data that show OCREVUS® (ocrelizumab) is a highly effective treatment option for people with relapsing-remitting multiple sclerosis (RRMS) who experienced a suboptimal response to their prior disease modifying therapy (DMT). and primary progressive MS (PPMS).

Treatment

Treatment FDA Approval Disease Clinical Trials

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

The Pharma Data

SEPTEMBER 2, 2020

New Phase III data from SAkuraStar and SAkuraSky studies demonstrate reduced severity of relapses with ENSPRYNG (satralizumab), recently FDA-approved as the first and only subcutaneous treatment for adults living with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD).

Clinical Trials

Clinical Trials Disease Treatment Therapies

BioSpace Global Roundup: Cevec Licenses AAV Program to Biogen

The Pharma Data

JANUARY 13, 2021

Germany-based Cevec Pharmaceuticals GmbH signed a licensing agreement with Biogen for the use of its proprietary ELEVECTA Technology for the manufacturing of adeno-associated virus (AAV) vectors for gene therapy applications. The deal will provide Biogen the rights to use the technology across their portfolio of gene therapy products.

Licensing

Licensing Licensing Clinical Trials Vaccine

2021 AAN highlight impact and breadth of expanding neuroscience portfolio

The Pharma Data

APRIL 8, 2021

FDA and global approvals for our groundbreaking therapies in SMA and NMOSD, Roche’s data at AAN reflect our continued commitment to meaningful therapeutic progress for people living with neurological disorders,” said Levi Garraway, M.D., Following U.S. Roche’s Chief Medical Officer and Head of Global Product Development.

Clinical Trials

Clinical Trials Trials Disease Nurses

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

In addition, over 170,000 people have been treated with OCREVUS, our first-in-class B-cell therapy, and we are incorporating years of clinical trial data and real-world evidence to optimise its potential to improve outcomes for patients with MS.”. Until the FDA approval of OCREVUS, there had been no FDA approved treatments for PPMS.

Clinical Trials

Clinical Trials Trials Disease FDA Approval

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

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

The Pharma Data

OCTOBER 26, 2020

Responses were observed in all primary tumor types and across all lines of prior therapy. Responses were observed in all primary tumor types and across all lines of prior therapy. This response developed as early as D43 and sustained at 6 months after initiation of therapy, consistent with the durable disease-control.

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

mRNA Cancer Vaccines and Therapies: An Overview

Trending Sources

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

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

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

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

BioSpace Global Roundup: Cevec Licenses AAV Program to Biogen

2021 AAN highlight impact and breadth of expanding neuroscience portfolio

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

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

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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