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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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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Eye on FDA

MARCH 7, 2024

Back in December 2023, FDA announced intention in the Federal Register and in a press release to form a new FDA Advisory Committee to be called the Genetic Metabolic Diseases Advisory Committee (GeMDAC).

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BioPharma Drive: Drug Pricing

MARCH 19, 2024

clearance of Lenmeldy, for a rare and inherited metabolic disease, triggers an additional payout related to Kyowa Kirin’s recent deal to acquire the once high-flying gene therapy developer.

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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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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. alone, according to the Centers for Disease Control and Prevention.

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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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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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BioPharma Drive: Drug Pricing

JULY 15, 2024

The company claims the results support exploring the possibility of an accelerated approval, citing flexibility by FDA officials in reviewing rare disease gene therapies.

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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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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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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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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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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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Drug Target Review

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

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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 ). All the data described above can be found in Sections 12 and 14 of the FDA-approved prescribing information (see here ). By Charles G.

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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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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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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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Drug Target Review

JANUARY 8, 2024

It is no mystery that as we age our health starts to deteriorate, and we become increasingly susceptible to diseases. Through years of scientific exploration and research, we now know there are several biological changes that make our bodies more susceptible to disease or injury, and we can target those with therapeutic interventions.

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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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KIF1A

MARCH 23, 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. We know that gene therapies are on the horizon, so it’s important to take note when groups make tangible progress.

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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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Eye on FDA

AUGUST 27, 2023

There are still many decisions and publicly available PDUFA dates with many novel treatments among them, including investigative treatments for sickle cell disease and ALS. AdComms and Approval Votes Are Up – In addition to NME approvals, there have been an increased number of FDA Advisory committees held to discuss new treatments.

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KIF1A

SEPTEMBER 30, 2023

Dylan Verden of KIF1A.ORG summarizes newly published KIF1A-related research and highlights progress in rare disease research and therapeutic development. KIF1A-Related Research In the rare disease space, a common and straightforward question is: How rare is the disease?

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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. This article explores the implications of this guidance, including new approaches for studies focused on cellular or gene therapies.

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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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FDA Law Blog: Drug Discovery

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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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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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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Drug Target Review

MAY 18, 2023

In sickle cell disease, however, this function is impaired, leading to a range of potential health consequences. The irregularly shaped and sticky red blood cells produced by the disease can obstruct small blood vessels, causing recurring painful attacks. Kutlar says the possibilities of this additional therapy are exciting.

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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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Drug Target Review

AUGUST 6, 2024

A number of areas represent significant opportunities for Antibody Drug Conjugates (ADCs) beyond oncology, leveraging their ability to deliver therapeutic agents specifically to diseased cells or tissues while minimising off-target effects. Oncology also offers high returns and the opportunity to address severe, life-threatening conditions.

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FDA Law Blog: Drug Discovery

DECEMBER 6, 2024

Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. Section #1: FDA Interactions Given the wide range of sponsors (i.e., Tobolowsky & Charles G. Raver & James E.

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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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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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