PLOS: DNA Science

OCTOBER 19, 2023

Last week DNA Science covered a setback in a clinical trial of a gene therapy for Duchenne muscular dystrophy (DMD). Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease.

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Therapies FDA Clinical Trials Disease 107

FDA Law Blog: Drug Discovery

MARCH 13, 2023

Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it is like to live with their condition, what symptoms most impact their lives, what goes into their decision about whether to participate in a clinical trial, and what kind of treatment effects would be most meaningful to them?

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Therapies Trials FDA Clinical Trials 98

BioPharma Drive: Drug Pricing

MARCH 6, 2025

Elsewhere, Novo started up an online pharmacy and the FDA cleared testing of a base editing therapy for Duchenne. Phase 3 studies for Amgen’s closely watched obesity drug were posted on a federal database.

Online Pharmacies 143

Online Pharmacies Pharmacy Trials FDA 143

FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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Therapies FDA FDA Approval Treatment 119

Advarra

NOVEMBER 14, 2024

In an era where clinical trials are increasingly global, it’s more imperative than ever to leverage international expertise. Data and safety monitoring boards (DSMBs), also known as data monitoring committees (DMCs), play a critical role in overseeing a clinical trial’s safety and efficacy. local standards of care).

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Clinical Trials Trials International Clinical Research 97

BioPharma Drive: Drug Pricing

FEBRUARY 20, 2024

The FDA approved the targeted therapy together with chemo for first-line metastatic lung cancer, while fresh trial results supported earlier use.

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Trials FDA Approval FDA Therapies 118

Eye on FDA

AUGUST 4, 2021

As more drugs are being approved, is FDA getting less advice than in the past? FDA maintains a vast network of outside advisors to provide input and counsel to the agency related to decisions on policy as well as product approvals. There are 31 advisory committees on matters ranging from food to medical devices to pediatric care.

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FDA Clinical Pharmacology FDA Approval Therapies 122

Codon

FEBRUARY 13, 2024

Tom Ireland writes about the companies and technologies that are reimagining phage therapy. Soon after its publication, scientists, journalists, and investors were revisiting ‘phage therapy’ as a promising alternative to our failing antibiotics. Read it on our website here. Illustration by David S. Fast forward to 2023.

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Therapies Virus DNA Clinical Trials 128

FDA Law Blog: Drug Discovery

JULY 17, 2024

Importantly, the Hub is intended to establish a new model within FDA, which leverages cross-Agency expertise in providing guidance and conducting reviews for products for rare disease populations. By Sarah Wicks & James E. Valentine & Frank J. those reviewed by the CDER Division of Rare Diseases and Medical Genetics).

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FDA Disease Clinical Pharmacology Science 105

New Drug Approvals

APRIL 2, 2025

2] [6] Suzetrigine is the first medication to be approved by the US Food and Drug Administration (FDA) in this new class of pain management medicines. [2] The FDA has long supported development of non-opioid pain treatment. acting director of the FDA’s Center for Drug Evaluation and Research.

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FDA Therapies Trials Treatment 62

PPD

JUNE 7, 2023

The number of pediatric patients diagnosed with nonalcoholic fatty liver disease (NAFLD) and nonalcoholic steatohepatitis (NASH) is on the rise, yet there are currently no approved therapies to treat NAFLD and NASH in adult or pediatric populations. As therapies for the treatment of NASH in adult patients go this year to the U.S.

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Trials Clinical Trials Clinical Research Disease 98

PPD

AUGUST 5, 2024

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success.

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Clinical Trials Trials Clinical Research Disease 98

Codon

NOVEMBER 3, 2024

The experience showed me that new therapies are needed not only to meet the targets laid out by the End TB Strategy but also to prevent drug resistance from negating the effectiveness of current therapeutics. A 1994 review of 14 prospective trials and 12 case-control studies revealed that the BCG vaccine reduced the risk of TB by 50 percent.

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Vaccine Trials Therapies Treatment 96

Drug Target Review

DECEMBER 13, 2024

2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates. These can include anti-tubulin compounds, potentially reducing side effects and improving quality of life.

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Therapies Treatment Clinical Trials Drugs 98

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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FDA Approval Treatment FDA DNA 97

FDA Law Blog: Drug Discovery

OCTOBER 11, 2023

Snow — On September 18, 2023, FDA published an updated, final iteration of guidance for immediate implementation entitled, “ Considerations for the Conduct of Clinical Trials of Medical Products During Major Disruptions Due to Disasters and Public Health Emergencies.” By James E. Valentine & Charles D.

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Clinical Trials Trials FDA Drug Development 64

FDA Law Blog: Drug Discovery

MARCH 26, 2024

Sasinowski — On March 21st, FDA announced the approval of the first nonsteroidal therapy for the treatment of Duchenne Muscular Dystrophy (DMD) (FDA press release available here ). The primary basis for approval, like other drugs for DMD, was based on a single placebo-controlled randomized trial. By Charles G.

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FDA Approval Therapies FDA Trials 59

Advarra

MARCH 20, 2023

The Food and Drug Administration (FDA) recently released new guidance regarding cellular and gene therapy products, one of which may significantly impact early-phase clinical trials of such products. The Benefits of the Umbrella Trial There are many potential benefits to the umbrella trial.

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Therapies FDA Trials Clinical Trials 52

Drug Target Review

JULY 4, 2023

These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Patients and caregivers also assess the benefits offered by different therapies, weighing the progression-free survival with their off-target effects. 3D rendering of Antibody Drug Conjugate Molecules.

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Small Molecule Drugs Clinical Pharmacology Trials 145

Conversations in Drug Development Trends

NOVEMBER 5, 2024

NC: I think the continued growth in cell and gene therapy research and the approvals we’re seeing for genetically-driven rare diseases. First, the beginning of the FDA’s START program, with the goal of accelerating the development of novel drugs and biological products for rare diseases.

Disease 52

Disease Clinical Trials Therapies Clinical Research 52

KIF1A

FEBRUARY 10, 2024

Read the Article Rare Roundup Bespoke Gene Therapy Consortium Publishes First ‘Playbook’ to Bring Rare Disease Programs to Clinic Gene-based therapy is still a new arena; even as scientists across the world work on gene therapies, a major question is how to best prepare these treatments for clinical trials and regulatory approval.

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Therapies Clinical Trials FDA Disease 111

Conversations in Drug Development Trends

NOVEMBER 27, 2023

Food and Drug Administration (FDA) announced its acceptance of the Biologics License Application (BLA) for exa-cel. In recognition of the unmet need and medical urgency for innovative therapies in the sickle cell space, the FDA granted exa-cel Priority Review, with a formal decision expected by December 8, 2023.

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Treatment FDA Clinical Trials Therapies 75

FDA Law Blog: Drug Discovery

MARCH 19, 2023

s Mark Tobolowsky co-authored the peer-reviewed article “ Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials ” in the recently published edition of Human Gene Therapy. Hyman, Phelps & McNamara, P.C.’s

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Therapies Clinical Trials Treatment Trials 98

The Pharma Data

FEBRUARY 24, 2022

Designation is supported by Phase II efficacy and safety data that will be presented at ATS 2022 Boehringer Ingelheim plans to study this novel investigational therapy in patients with progressive fibrosing interstitial lung diseases. The FDA Breakthrough Therapy designation is supported by data collected to date.

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Therapies FDA Clinical Trials Disease 52

Chemical Biology and Drug Design

OCTOBER 10, 2023

Zidovudine, didanosine, and stavudine are FDA-approved NRTIs, while nevirapine, efavirenz, and delavirdine are FDA-approved NNRTIs. Several agents are in clinical trials, including apricitabine, racivir, elvucitabine, doravirine, dapivirine, and elsulfavirine.

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FDA Approval Clinical Trials FDA Virus 100

Alta Sciences

FEBRUARY 19, 2025

Food and Drug Administration (FDA) issued two guidance documents outlining the necessary evaluations during the clinical development of oligonucleotide therapeutics: Clinical Pharmacology Considerations for the Development of Oligonucleotide Therapeutics and Nonclinical Safety Assessment of Oligonucleotide-Based Therapeutics .

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Drug Development Clinical Pharmacology Immune Response Drugs 52

Fierce BioTech

SEPTEMBER 20, 2024

1 2 The diagnosis of cancer in children may be delayed due to the non-specific nature of t | Navigate current legislation and FDA guidance for pediatric oncology clinical trials with expert guidance from WCG. Although childhood cancer is rare, it is the leading cause of disease related death in children.1

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Clinical Trials Trials Therapies Research 52

Advarra

NOVEMBER 29, 2022

The field of cell and gene therapies (CGT) is constantly evolving, and there has been significant progress in this area of research. However, despite the promise of these therapies, the regulations governing them lag the science, which in turn hinders the clinical translation of these novel medicines.

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Therapies Clinical Trials FDA Trials 52

PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. DEB has been a candidate for a gene therapy since 2002.

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FDA Approval Therapies FDA Disease 52

The Premier Consulting Blog

OCTOBER 21, 2024

Accelerated approval is an expedited regulatory pathway designed to hasten the availability of drugs (including biologics) that treat serious conditions, offer advantages over existing therapies, and address unmet medical needs. In essence, these trials are the final step that turns provisional approval into full approval.

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Trials FDA Clinical Trials Disease 52

The Pharma Data

MAY 27, 2022

Novartis today announced the US Food and Drug Administration (FDA) has granted accelerated approval for Kymriah ® (tisagenlecleucel) for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of systemic therapy.

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FDA Approval Therapies FDA Treatment 52

Antidote

JULY 9, 2024

Though clinical trials are often discussed in the context of medications and therapies, the devices that administer these medications must also be approved by the FDA before they can be released to market.

Clinical Trials 52

Clinical Trials Trials FDA Therapies 52

Drug Target Review

FEBRUARY 21, 2024

Stem cell transplants have saved patients’ lives time and time again, which led us to launch our own Stem Cell Transplant and Cellular Therapy Program. Regarding CAR-T cell therapy, could you highlight the promising findings that have been identified in the early stages of drug discovery, as well as potential challenges?

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Therapies Treatment Vaccine FDA Approval 64

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

KIF1A

APRIL 25, 2024

She is scheduled to be the 2nd patient in the world to receive ASO therapy for KAND. March 2023 – We met with n-Lorem and found out Susannah’s ASO (1st KAND patient to receive ASO therapy for KAND) would work for Sloane. February 2024 – n-Lorem submitted an IND (investigational new drug) to the FDA for Sloane.

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Therapies FDA Approval FDA Treatment 52

National Institute on Drug Abuse: Nora's Blog

MARCH 6, 2025

Advancing reduction of drug use as an endpoint in addiction treatment trials astewart Thu, 03/06/2025 - 09:59 Nora's Blog March 18, 2025 Image Getty Images/ SolStock This blog was also published in the American Society of Addiction Medicine (ASAM) Weekly on March 18, 2025.&

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Treatment Trials Clinical Trials Drugs 69

PLOS: DNA Science

OCTOBER 12, 2023

In the final chapter of my 2012 book The Forever Fix: Gene Therapy and the Boy Who Saved It , I predicted that the technology would soon expand well beyond the rare disease world. Gene therapy clearly hasn’t had a major impact on health care, offering extremely expensive treatments for a few individuals with rare diseases.

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Therapies Immune Response DNA Virus 69