FDA Law Blog: Drug Discovery

MAY 19, 2024

By Sarah Wicks — On May 21, 2024, the EveryLife Foundation for Rare Diseases (ELF) will host a Scientific Workshop at the National Press Club in Washington, D.C. aimed at identifying and characterizing the challenges in developing therapies for ultra-rare diseases and conditions that affect exceedingly small populations.

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FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” There are hundreds of known genetic metabolic diseases, most of which are rare and carry significant morbidity.

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BioPharma Drive: Drug Pricing

SEPTEMBER 18, 2023

Three years after gaining European approval, Libmeldy is now under U.S. review with a deadline set for March.

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Drug Target Review

OCTOBER 16, 2023

How can PDOs revolutionise drug discovery and deepen our understanding of disease? Patient-derived organoids (PDOs) are proliferative 3D cell structures derived from tissue samples of both healthy and diseased tissue. Improved translatability to the patient and the disease state.

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BioPharma Drive: Drug Pricing

APRIL 1, 2024

The clearance of Voydeya as an add-on therapy for an uncommon blood disease validates a bet Alexion made on original developer Achillion five years ago.

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BioPharma Drive: Drug Pricing

JULY 3, 2024

Multibillion-dollar buyouts from Bristol Myers Squibb and Gilead could yield new drugs for brain and liver diseases, while a new cell therapy may reach market.

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The Pharma Data

AUGUST 9, 2021

Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients 1 year of age and older with late-onset Pompe disease. “Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives,” said Janet Maynard, M.D.,

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FDA Law Blog: Drug Discovery

JUNE 12, 2024

Sasinowski — Hyman, Phelps & McNamara (HPM) would like to congratulate the 7 rare disease programs selected for the inaugural class of the FDA’s “Support for clinical Trials Advancing Rare disease Treatment” (START) pilot program. HPM is proud to aid 5 of the 7 selected participants for the START pilot program.

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PLOS: DNA Science

MARCH 28, 2024

A new drug has entered the arsenal against Duchenne muscular dystrophy (DMD), a genetic disease that affects boys and is challenging to treat. FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. Muscle makes up about 40 percent of body weight. million DNA bases.

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Agency IQ

FEBRUARY 12, 2024

BY RACHEL COE, MSC | FEB 7, 2024 10:21 PM CST The Bespoke Gene Therapy Consortium A collaborative effort, the Bespoke Gene Therapy Consortium (BGTC) was launched in October 2021 to “accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease.”

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BioPharma Drive: Drug Pricing

JUNE 22, 2023

The conditional clearance for Sarepta’s Elevidys in 4- and 5-year-olds is a milestone for research into the deadly disease, and raises the stakes of an ongoing trial that could prove how well it works.

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The Pharma Data

OCTOBER 14, 2021

Roche announced that gantenerumab, an anti-amyloid beta antibody developed for subcutaneous administration, has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of people living with Alzheimer’s disease (AD).

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Broad Institute

SEPTEMBER 13, 2023

Developing diagnostics and treatments By Rose Circeo September 13, 2023 Breadcrumb Home Developing diagnostics and treatments At the Broad Institute, researchers are revealing deep biological insights about the causes of disease, which are leading to new ways to monitor, diagnose, and treat patients with greater precision.

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Drugs.com

OCTOBER 31, 2023

30, 2023 -- Patients with sickle cell disease may soon have two new treatments to try. Food and Drug Administration advisory committee will weigh the merits of a new gene therapy for the painful, inherited. MONDAY, Oct. On Tuesday, a U.S.

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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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BioPharma Drive: Drug Pricing

FEBRUARY 16, 2024

The agency will decide by June 21 whether to enable more patients with the disease to receive Elevidys, and won’t convene a group of outside experts beforehand.

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FDA Law Blog: Drug Discovery

NOVEMBER 2, 2023

Lewis, Senior Regulatory Device & Biologics Expert — On October 20, 2023, FDA announced the availability of the final guidance authored by CBER titled “Voluntary Consensus Standards Recognition Program for Regenerative Medicine Therapies.” Tobolowsky & Richard A. It finalized a draft guidance published in 2022.

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BioPharma Drive: Drug Pricing

AUGUST 15, 2023

The agency had paused testing after a patient death, but is now permitting more types of bridging treatment to help keep participants’ disease at bay.

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The Pharma Data

JUNE 1, 2022

The United States Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to efanesoctocog alfa (BIVV001) for the treatment of people with hemophilia A, a rare and life-threatening bleeding disorder, based on data from the pivotal XTEND-1 P hase 3 stud y.

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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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Agency IQ

JUNE 26, 2023

FDA quietly kicks off process that could inform future rare disease regulatory reforms Reforms buried within the FY2023 omnibus spending bill require the development of a new report looking at the regulatory processes of the U.S. Meanwhile, in the EU rare diseases are defined using a different set of criteria. In the U.S.,

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Drug Target Review

AUGUST 29, 2024

What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies? MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs.

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The Pharma Data

DECEMBER 15, 2020

Food and Drug Administration (FDA) granted Novartis ’ experimental drug iptacopan Breakthrough Therapy designation in paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder. Breakthrough Therapy designation will expedite the development and review of iptacopan for this indication.

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Cytel

APRIL 10, 2024

Orphan drug designation is a regulatory status granted to pharmaceuticals developed for the treatment of rare diseases. It provides incentives to encourage the research, development, and approval of therapies targeting small patient populations.

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Drug Channels

APRIL 4, 2022

Rare Disease Innovation & Partnering Summit. Don’t miss the Rare Disease Innovation & Partnering Summit, coming up May 17-19, 2022 in Boston, MA. Hybrid Event May 17-19, 2022 | Boston, MA www.informaconnect.com/rare. Visit www.informaconnect.com/rare for further details and to register.

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Fierce BioTech

SEPTEMBER 20, 2024

Although childhood cancer is rare, it is the leading cause of disease related death in children.1 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.

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The Premier Consulting Blog

MARCH 7, 2024

The FDA’s January 2020 guidance, Chemistry, Manufacturing and Control (CMC) [1] Information for Human Gene Therapy Investigational New Drug Applications (INDs), outlines the analytical methods that define the quality, safety and efficacy of gene therapy therapeutics.

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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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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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The Pharma Data

OCTOBER 9, 2021

Food and Drug Administration (FDA) for the treatment of people living with Alzheimer’s fever ( Notice). About gantenerumab and its clinical program Gantenerumab is an investigational IgG1 antibody designed to bind to aggregated forms of beta-amyloid and remove brain amyloid plaques, a pathological hallmark of Alzheimer’s disease (AD).

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Advarra

MARCH 10, 2023

Having a one in four chance of developing sickle cell disease (SCD) is uniquely, and overwhelmingly, familiar to the African American population. Considered a rare disease, research in finding better therapies – and possibly even a cure – for SCD may be competing for funding with conditions impacting broader populations.

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Drug Target Review

JUNE 3, 2024

How does COUR Pharmaceuticals’ immune-modifying nanoparticle platform differ from traditional approaches to treating immune-mediated diseases? Essentially, our CNPs reprogramme the immune system by restoring balance and returning the body to a homeostatic state, offering a precise approach to treating immune-mediated diseases.

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The Pharma Data

APRIL 7, 2022

This unprecedented CMS decision effectively denies all Medicare beneficiaries access to ADUHELM ® (aducanumab-avwa), the first and only FDA-approved therapy in a new class of Alzheimer’s drugs. ADUHELM is indicated for the treatment of Alzheimer’s disease. ADUHELM is indicated for the treatment of Alzheimer’s disease.

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Fierce BioTech

SEPTEMBER 11, 2023

Aligning cross-functional teams across the organization to ensure medical, marketing, and market access are all in lock-step is even more crucial when launching a therapy into a rare disease market. Click here to login. Listing Image HerspiegelConsulting_LogoListingNew_10262023.png On Demand Start Date Thu, 10/26/2023 - 11:00

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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. The treatment is being researched by scientists collaborating from American Gene Technologies, the Laboratory of Immunoregulation and the National Institute of Allergy and Infectious Diseases. Conor Kavanagh.

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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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KIF1A

FEBRUARY 10, 2024

Each week, Dr. Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. This highlights the heterogeneity of KAND, and the importance of identifying KIF1A mutations in other rare disease groups.

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