FDA Law Blog: Drug Discovery

MARCH 26, 2024

Emily Freilich, Director of the Division of Neurology 1 in the Office of Neuroscience in CDER noted in FDA’s press release, Duvyzat is the first nonsteroidal DMD therapy approved regardless of genetic mutation. The primary basis for approval, like other drugs for DMD, was based on a single placebo-controlled randomized trial.

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The Pharma Data

DECEMBER 15, 2020

FDA Approves Klisyri (tirbanibulin) for the Treatment of Actinic Keratosis on the Face or Scalp. NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, today announced that the U.S. BUFFALO, N.Y.,

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The Pharma Data

FEBRUARY 25, 2022

Several FDA-approved drugs – including for type 2 diabetes, hepatitis C and HIV – significantly reduce the ability of the Delta variant of SARS-CoV-2 to replicate in human cells, according to new research led by scientists at Penn State. Pfizer’s COVID-19 therapy Paxlovid, for example, targets Mpro.

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The Pharma Data

NOVEMBER 24, 2020

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

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The Pharma Data

DECEMBER 17, 2020

There will be a brand-new treatment for actinic keratosis on the market next year, with Tuesday’s FDA approval of Athenix’s Klisyri® (tirbanibulin). The FDA approval of Klisyri is a significant milestone for Athenex. The product launch is expected to take place in March of 2021. Athenex, Inc.

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ASPET

OCTOBER 12, 2023

To maintain cadence with looming threats in a prolonged field care environment, the broader medical countermeasure (MCM) enterprise must adopt new strategies for CBRN-addressing drug development.

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The Pharma Data

NOVEMBER 12, 2021

Treatment is First FDA-Approved Option Patients Can Take Regardless of Previous Therapies. Food and Drug Administration approved Besremi (ropeginterferon alfa-2b-njft) injection to treat adults with polycythemia vera, a blood disease that causes the overproduction of red blood cells. Today, the U.S. Related Information.

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Broad Institute

JULY 17, 2024

Srijit Seal, a visiting scholar at the Carpenter-Singh Lab in the Broad's Imaging Platform , trained multiple predictive machine learning models to identify chemical and structural drug features likely to cause toxic effects in humans.

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The Pharma Data

MARCH 15, 2022

The FDA regularly takes steps to help guide industry through the development process for generic drug products, including combination products, such as MDIs, that consist of a drug and a device. The FDA, an agency within the U.S. Related Information.

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FDA Law Blog: Biosimilars

JUNE 27, 2023

James was only one of five life sciences attorneys selected and the only food and drug lawyer to make the list. Amongst his accomplishments, Law360 considered the role James has played in leveraging little-used pathways to FDA approval for often first-ever drugs to treat rare diseases (e.g.,

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Drug Target Review

JANUARY 8, 2024

We are at the forefront of drug development in an area of research called cellular rejuvenation, which is an approach that has the potential to address many diseases of ageing by restoring aged and injured cells to a more youthful and resilient state. Most recently, Jerry was President and CEO for Neos Therapeutics, Inc.,

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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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Pharmaceutical Development Group

DECEMBER 19, 2021

Effective use of Real World Data (RWD) and Real World Evidence (RWE) can reduce costs of Pharmaceutical Drug and Biologic Products and Process Development, Drug Development Services , expedite a FDA Pre-Submission Review, and lead to FDA Approval. Author Information William E. Spanogle, Ph.D.

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The Pharma Data

JULY 8, 2021

Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product.

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Pharmaceutical Development Group

JANUARY 7, 2022

Effective and consistent use and application of Data Standards can reduce costs of Pharmaceutical Drug and Biologic Products and Process Development, Drug Development Services, 505(b) NDA, IND Consulting , NDA Consulting, BLA Consulting , and effective FDA Pre-Submission collectively resulting in FDA Approval.

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PLOS: DNA Science

SEPTEMBER 5, 2024

A Brief History of Targeted Cancer Drugs In 1978, FDA approved the first targeted cancer drug, tamoxifen. The FDA approved Herceptin , targeting a different receptor (HER2) in breast cancer cells, in 1998. Keytruda illustrates more recently approved cancer drugs.

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FDA Law Blog: Drug Discovery

OCTOBER 11, 2023

In the case of trials for orphan drugs, this is particularly concerning because there is often already difficulty in interpreting clinical trial results to establish effectiveness due to the common challenges inherent in rare disease drug development.

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Advarra

AUGUST 16, 2022

Orphan drugs have historically faced a number of barriers, such as limited research and development (R&D) investment due to an expected lack of profitability as well as challenges in clinical trial design and recruitment. Before 1983, only 38 orphan drugs had received U.S. Food and Drug Administration (FDA) approval.

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New Drug Approvals

APRIL 2, 2025

1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] 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] 6 February 2025.

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The Pharma Data

DECEMBER 16, 2020

Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to eight FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories.

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KIF1A

JUNE 3, 2023

Read the Paper Rare Roundup Cautious Optimism for Newly Approved Drug for Rare Form of Genetic ALS When is there enough evidence for a drug that we can call it a treatment for a disease? It’s important to show that a drug works as intended to improve the lives of patients, and does so with reasonable safety.

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Drug Target Review

MAY 1, 2024

What specific patient populations are benefiting the most from these developments? To date, the 11 FDA-approved ADCs have shown benefit to patients across multiple solid and hematological malignancies with approvals in more than 20 indications. Dr Moore received a PhD in molecular genetics from the University of Glasgow.

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The Pharma Data

JANUARY 19, 2021

Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to eight FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories.

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The Pharma Data

NOVEMBER 30, 2020

Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to eight FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories.

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The Pharma Data

OCTOBER 27, 2020

Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to eight FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories.

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Drug Target Review

SEPTEMBER 19, 2023

In addition, these models at the current state are better for short term tests, over a week or two and cannot be used for acquiring data from long term drug exposures. Miniature organs as test beds for new drugs Organoids are miniature human organs with clusters of cells derived from stem cells and mimic specific tissues.

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PerkinElmer

JANUARY 7, 2022

The pharmaceutical industry is under huge pressure to address the high attrition rates in drug development. With around 90% of candidates failing during clinical development, 1 the process is not only long and risky, but also expensive for those involved. References Hingorani, A.D., Sci Rep 9, 18911 (2019).

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Drug Target Review

MAY 27, 2024

At a base-layer, Caris’ comprehensive molecular profiling that we perform for patients today can identify predictive biomarkers for current FDA-approved ADCs. What role do ADCs play in Caris’ strategy for targeted cancer therapy, particularly in terms of patient-specific treatment plans? Examples include HER2-low and FOLR1.

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Codon

NOVEMBER 3, 2024

As an undergraduate biology student, I spent some time in a TB lab working on antibiotic resistance — a growing concern for drug developers. Food and Drug Administration (FDA)-approved drug library, some of which inhibit E. coli bacteria from growing. However, hope prevails.

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Conversations in Drug Development Trends

MAY 30, 2024

This trend is on the rise despite recent disappointments with clinical trial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. The shared placebo design is another approach to accelerating drug development in ALS research.

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The Connected Lab

APRIL 8, 2020

For patients suffering from an illness with no approved treatment, the wait can be unnerving. To this day, more than 400 million people suffer from rare diseases and 95% of rare diseases lack an FDA approved treatment 3. How is AI being applied in Drug Discovery efforts?

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The Pharma Data

JANUARY 25, 2021

Orphan drug designation provides eligibility for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval. About VLX-1005.

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Conversations in Drug Development Trends

FEBRUARY 26, 2024

However, incorporating the patient experience into your design is one of the best ways to ensure your drug development program is a commercial success. It plays a significant role in patient recruitment and retention, both of which are vital to maintaining timelines, decreasing program costs, and gaining market approval.

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NIH Director's Blog: Drug Development

JUNE 14, 2016

Several years ago, the Food and Drug Administration (FDA) recommended that drug developers take special care to show that potential drugs to treat diabetes don’t adversely affect the cardiovascular system [1].

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The Premier Consulting Blog

NOVEMBER 12, 2023

The FDA is actively supporting this increased AI/ML integration in a variety of ways, including: Establishing the CDER AI Steering Committee (AISC) to provide guidance and oversight on AI/ML in drug development. Developing a framework for AI/ML-based devices to ensure that these devices are safe and effective.

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Alta Sciences

FEBRUARY 8, 2024

Utilizing 10 state-of-the-art simulators available in-house at our Montréal clinical facility (with the capacity for more than 20), we are equipped to measure a range of studies; from impairment in cognition and comparing compounds to assessing the impact on new formulations have on impairment.

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Vial

DECEMBER 5, 2023

It has become a fundamental tool for researchers to explore the complexities of genetic information and conduct genetic-informed drug development. Sponsors have used NGS to screen patients for clinical trial eligibility and patient stratification , expanded CDx development, and comprehensive genomic profiling (CGP).

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