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. CBRN MCMs).

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Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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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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Drug Target Review

JULY 4, 2023

Over the last two decades, an increasing number of Antibody Drug Conjugate (ADC) therapeutics have been approved for oncology indications. These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. Antibody drug conjugated with cytotoxic payload.

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FDA Law Blog: Drug Discovery

MAY 1, 2024

proudly announces the return of attorney Sarah Wicks to its drug development and compliance group. Sarah brings a wealth of experience and a proven track record of advising innovative drug and biologics companies through the intricate landscape of product development and commercialization. Valentine, HPM Director.

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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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The Pharma Data

DECEMBER 17, 2020

Food and Drug Administration (FDA) had several approvals this week. Food and Drug Administration (FDA) has approved belimumab (Benlysta®), the first-ever treatment for adults with lupus nephritis (LN) who are currently receiving standard therapy. The FDA approval of Klisyri is a significant milestone for Athenex.

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FDA Law Blog: Drug Discovery

MAY 19, 2024

aimed at identifying and characterizing the challenges in developing therapies for ultra-rare diseases and conditions that affect exceedingly small populations. 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.

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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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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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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

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The Premier Consulting Blog

JUNE 18, 2023

This has opened new opportunities in pharmaceutical drug development, such as the ability to evaluate large complex databases and to integrate information in useful ways. One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases.

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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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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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KIF1A

SEPTEMBER 30, 2023

Read the Guide FDA Launches Pilot Program to Help Further Accelerate Development of Rare Disease Therapies One frustrating aspect of traditional drug development, especially for rare disease communities, is the tempo of regulatory decisions on potential drugs.

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Drug Target Review

DECEMBER 5, 2024

Viral vectors have been crucial in transforming the gene therapy landscape due to their natural ability to infect cells. 1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2.

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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.” Drug development for these conditions has unique and complex challenges, therefore few treatments are available to patients.”

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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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Drug Target Review

JANUARY 8, 2024

Contrary to popular belief, ageing is not caused by just random wear and tear of our bodies over time but is instead caused by a discrete set of biological mechanisms that we now better understand and can target with therapies. a clinical-stage private biopharma company developing therapies for neurological and psychiatric diseases.

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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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Drug Target Review

SEPTEMBER 22, 2023

Organoid technologies are becoming an invaluable solution for preclinical research, with the ability to augment the development of personalised medicine, drug discovery and gene therapies. 5 Organoids are recognised as New Alternative Methods (NAMs) in drug development.

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FDA Law Blog: Drug Discovery

JANUARY 4, 2023

Brevig, Senior Regulatory Device and Biologics Expert — On December 7, 2022, FDA’s Center for Biologics Evaluation and Research (CBER) and the Office of Tissues and Advanced Therapies (OTAT) held a town hall to answer questions related to cell therapy and tissue-engineered products chemistry, manufacturing, and contr­­ols (CMC).

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The Premier Consulting Blog

JUNE 26, 2023

1 With their poorly understood natural histories, phenotypic heterogeneity, and diverse clinical manifestations, rare cancers pose challenges to drug development and represent a significant unmet need in oncology. Rare cancers account for 25-30 percent of all new cancer diagnoses and 25 percent of cancer deaths.

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The Pharma Data

MAY 17, 2021

Acting FDA Commissioner. During this global pandemic, the FDA worked to protect the American public by using every tool at its disposal to quickly review and act on new therapies for COVID-19. Conducting clinical trials to see if a drug is safe and effective can be a very time-consuming process. .

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The Pharma Data

AUGUST 4, 2020

Novartis is celebrating the success of its one-time CAR T therapy Kymriah (tisagenlecleucel) in patients with relapsed or refractory (r/r) follicular lymphoma (FL), revealing that the therapy met its primary its primary endpoint of complete response rate (CRR) in Phase 2 trials, according to an independent review committee.

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FDA Law Blog: Drug Discovery

FEBRUARY 28, 2024

This is the third withdrawal of an accelerated approval FDA has performed, following Avastin in 2011 and Makena in 2023. Notably, this decision marks the first use of the new expedited procedures for withdrawal of an accelerated approval that were enacted in the Food and Drug Omnibus Reform Act of 2022 (FDORA).

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The Pharma Data

AUGUST 5, 2020

. Novartis has revealed positive results from a phase 2 study of its CAR-T therapy Kymriah (tisagenlecleucel) in patients with relapsed or refractory follicular lymphoma. The therapy is a one-time treatment created individually for each patient using their own T cells.

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FDA Law Blog: Biosimilars

OCTOBER 10, 2023

(“HP&M”), the largest FDA-dedicated law firm, is pleased to announce that Julie Beitz, M.D. has joined the firm as a Principal Drug Regulatory Expert. Dr. Beitz served in leadership positions in FDA’s Center for Drug Evaluation and Research for nearly three decades. Ellis Unger who joined HP&M in February 2022.

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The Pharma Data

NOVEMBER 20, 2020

(NASDAQ: REGN) today announced that the antibody cocktail casirivimab and imdevimab administered together (formerly known as REGN-COV2 or REGEN-COV2), a therapy currently being investigated for use in COVID-19 , has received Emergency Use Authorization (EUA) from the U.S. Food and Drug Administration (FDA). Schleifer, M.D.,

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KIF1A

JANUARY 13, 2024

Aditi Falnikar’s presentation from our 2023 KAND Conference Read the Article Rare Roundup FDA issues final guidance on rare disease drug development As we continue to explore new therapeutic avenues for treating KAND, it is crucial to think about the regulatory steps those therapies will face.

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The Premier Consulting Blog

APRIL 18, 2024

Development of cell and gene therapies is growing rapidly, given the major advances in genomic technologies and increasing scientific understanding of genetic regulation and immunology. As in all nonclinical drug development programs, the goal is to identify potential safety issues in humans. Is fit-for-purpose?

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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. 7 Recent clinical studies highlight promising developments in c-MET-targeting ADCs for NSCLC.

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The Pharma Data

MAY 17, 2021

Sponsors using master protocols to test new drugs and therapies for treating or preventing COVID-19 should base their analyses on comparisons between control arm participants who were concurrently randomized, the FDA advised in a final guidance released yesterday.

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FDA Law Blog: Drug Discovery

DECEMBER 15, 2024

Valentine We recently published the first part of our review of FDAs draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. If you make a tissue product you may produce one lot per patient where a gene therapy may only need a handful of lots through Phase 3.

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The Pharma Data

JANUARY 13, 2021

14, 2021 /PRNewswire/ — MindMed (NEO: MMED), (OTCQB: MMEDF), (DE: MMQ), a leading psychedelic medicine biotech company today announced the addition of Robert Barrow , an accomplished pharmaceutical executive, as Chief Development Officer. We are excited to attract such top tier talent from the psychedelic drug development community.

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The Pharma Data

JANUARY 4, 2021

5, 2021 /RPNewswire/ — Last December 25 , the anti-tumor drug candidate Chiauranib, developed by Shenzhen Chipscreen Biosciences Co., In addition, a Pre-IND communication with the FDA for clinical trial of Chiauranib against SCLC in US has been conducted. About the Breakthrough Therapy Designation.

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The Pharma Data

JANUARY 25, 2021

.–( BUSINESS WIRE )– Veralox Therapeutics, a biotechnology company developing first-in-class small molecule therapeutics that treat the underlying pathologies of diseases with significant unmet medical needs, today announced that the U.S. Boxer, PhD, Chief Operating Officer of Veralox Therapeutics. About VLX-1005.

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