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

OCTOBER 25, 2024

How does precision neuroscience differ from traditional approaches in the treatment of neurodegenerative diseases, and what advantages does it offer? Traditionally, Alzheimer’s disease is diagnosed by clinical examination, medical history and cognitive testing.

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Drug Target Review

AUGUST 29, 2024

However, these treatments will eventually fail for the majority of patients, so there is a strong need for better CAR therapies. How might the CD5 knockout approach impact the treatment of other types of cancers beyond T-cell lymphoma?

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Conversations in Drug Development Trends

FEBRUARY 28, 2023

95% of rare diseases don’t have a treatment approved and won’t anytime soon. Miraculously, there was an approved treatment. This treatment only exists because a small charity in the ’70s donated the funds needed for the researcher to discover it. Uplifting Athletes (UA) is on a mission to change that. It saved my life.”

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Drug Target Review

NOVEMBER 6, 2023

Advances in research and development and the utilisation of combination treatment strategies are revolutionising the haematology care landscape, but more must be done to address patients’ individual needs. 2 It is this complexity that necessitates powerful, targeted combination therapies.

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The Pharma Data

DECEMBER 17, 2020

18, 2020 /PRNewswire/ — Camurus AB (NASDAQ STO: CAMX) announced today that the Swiss agency for therapeutic products, Swissmedic, has approved weekly and monthly Buvidal ® prolonged release buprenorphine for the treatment of opioid dependence in adults and adolescents from 16 years of age. LUND, Sweden , Dec. About Buvidal.

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Broad Institute

NOVEMBER 4, 2024

By Lucy Mutz November 4, 2024 Credit: Juliana Sohn Kasper Lage is managing director of the Novo Nordisk Foundation Center for Genomic Mechanisms of Disease. The overall goal is to translate genetic findings into biological mechanisms underlying disease that could be targeted with new medicines.

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Advarra

JULY 26, 2022

The 2018 Farm Bill. The 2018 Farm Bill removed hemp from the Controlled Substance Act. Importantly, the 2018 Farm Bill preserved FDA authority to regulate products with cannabis or cannabis-derived compounds under the Federal Food, Drug, and Cosmetic (FD&C) Act and Section 351 of the Public Health Service Act.

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PLOS: DNA Science

MARCH 21, 2024

million price tag for the one-time infusion, and for the older siblings who contributed to developing the gene treatment, but were too sick to receive it. The history of gene therapy for MLD is compelling – DNA Science covered it for Rare Disease Day in 2021, here. She passed away in 2013.

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The Pharma Data

DECEMBER 4, 2020

Alzheimer Disease Tied to Adverse Financial Events. 4, 2020 — Alzheimer disease and related dementias (ADRDs) are associated with adverse financial events years prior to clinical diagnosis, according to a study published online Nov. Professional. FRIDAY, Dec. 30 in JAMA Internal Medicine. Lauren Hersch Nicholas, Ph.D.,

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The Pharma Data

APRIL 17, 2021

Biogen’s expansion in China now includes treatment options approved for relapsing MS and spinal muscular atrophy. today announced that China’s National Medical Products Administration (NMPA) has approved TECFIDERA ® (dimethyl fumarate) for the treatment of relapsing multiple sclerosis (MS).

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New Drug Approvals

OCTOBER 12, 2024

Alzheon licensed ALZ-801 from NeuroChem and is developing it for Alzheimer’s disease. ALZ-801 is an advanced and markedly improved candidate for the treatment of alzheimer’s disease. 2018; 32(9): 849–861. [2]. 2018 Mar;57(3):315-333. 2007 ; Kocis et al., 2016 ; Abushakra et al., Hey JA, et al. Clin Pharmacokinet.

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The Pharma Data

NOVEMBER 2, 2020

The World Health Organization’s (WHO) Global TB Programme welcomes the results from an important study on shortened treatment for drug-susceptible tuberculosis (TB) in children, presented at the 51 st virtual Union World Conference on Lung Health. Side-effects related to treatment were few, and similar across both groups.

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The Pharma Data

FEBRUARY 8, 2021

Janssen Announces Treatment with ERLEADA ® (apalutamide) Significantly Improved Overall Survival in Patients with Metastatic Castration-Sensitive Prostate Cancer. Based on these data, ADT alone should no longer be considered sufficient for patients with advanced, castration-sensitive disease.”. About ERLEADA ® (apalutamide).

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Drug Target Review

NOVEMBER 3, 2023

The findings may have implications for our understanding of gastrointestinal diseases and endocrine disorders, as well as pancreatic development and diabetes. Further research This discovery may have implications for understanding gastrointestinal diseases and endocrine disorders. 2018 February 8 [2023 November 1];172(4):650-65.

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The Pharma Data

JANUARY 11, 2021

-based subsidiary of Terumo and a global neurovascular company, announced today the FDA Approval of the PMA Supplement for the WEB 17 System, a new addition to the WEB Aneurysm Embolization System for the treatment of intracranial wide neck bifurcation aneurysms. The WEB System received its first PMA approval in late 2018.

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Drug Target Review

NOVEMBER 7, 2024

Introduction Therapeutic antibodies have proven to be indispensable medicines for addressing the most debilitating diseases. Agonist antibodies in immunological diseases Agonist antibodies targeting immune checkpoint pathways are increasingly recognised and investigated for their potential in immunological diseases.

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Drug Target Review

FEBRUARY 28, 2024

Over the past 25 years, T-cell therapies have gained significant ground in the treatment of cancer. Human γδ TCR repertoires in health and disease. Trends Immunol 39(6):446-459 (2018). Nat Commun 9(1):1760 (2018). J Front Immunol 9:1409 (2018). References Fichtner A, Ravens S, Prinz I. Cells 9(40):800 (2020).

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The Pharma Data

DECEMBER 7, 2020

based clinical trials investigating interventions for high blood pressure between 2009-2018, just 52 were exclusively in Black people. While the numbers grew over that decade – from 3.9% (3 of 77 trials) in 2009 to 6.2% (9 of 146) in 2018 – the increase was not enough to be statistically significant.

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The Pharma Data

NOVEMBER 24, 2020

25, 2020 /CNW/ – On August 21, 2020 , Health Canada approved Lynparza® (olaparib), for the treatment of adult patients with deleterious or suspected deleterious germline and/or somatic BRCA or ATM- mutated metastatic castration-resistant prostate cancer (mCRPC) who have progressed following prior treatment with a new hormonal agent (NHA).

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Conversations in Drug Development Trends

FEBRUARY 22, 2023

Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.

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The Pharma Data

DECEMBER 3, 2020

3, 2020 (American Heart Association News) — Your chances of dying from heart disease or stroke are higher if you live in a county considered socially vulnerable due to factors such as poverty, crowded housing and poor access to transportation, new research shows. THURSDAY, Dec. More than 1 in 3 U.S.

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New Drug Approvals

SEPTEMBER 29, 2024

gingivalis has been called a risk factor for Alzheimer’s disease ( Kanagasingam et al., 2018 ; Ding et al., gingivalis is associated with cardiovascular disease. Both were reversed by treatment with COR388 ( 2020 AAIC abstract ). Periodontitis has been linked epidemiologically to cognitive impairment, and P.

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The Pharma Data

NOVEMBER 18, 2021

Eli Lilly and Company blazoned that registration is now open for the CHALLENGE-MIG clinical trial, the first and only head-to- head trial comparing twoanti-calcitonin gene- related peptide (CGRP) drugs for the preventative treatment of episodic migraine in grown-ups.1 gepants) may help people achieve the pretensions that count most to them.

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The Pharma Data

JUNE 28, 2021

Food and Drug Administration (FDA) and a marketing authorization application (MAA) to the European Medicines Agency (EMA) seeking approval of the novel combination of the cancer treatments copanlisib and rituximab. submission is for the treatment of patients with relapsed indolent B-cell non-Hodgkin’s lymphoma (B-iNHL). Fields, M.D.,

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The Pharma Data

SEPTEMBER 18, 2020

Based on this recommendation, a final decision regarding approval of Tecentriq in combination with Avastin in this disease setting, along with the full details of the approved indication, is expected from the European Commission in the near future. 0.76; p<0.0001), compared with sorafenib. RECIST v1.1).

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The Pharma Data

DECEMBER 3, 2020

The TAK-620-303 (SOLSTICE) trial (NCT02931539) is a multicenter, randomized, open-label, active-controlled trial comparing eight weeks of treatment with either maribavir or investigator assigned treatment (IAT) in transplant recipients with CMV infection refractory or resistant to existing antiviral treatments (i.e.,

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The Pharma Data

DECEMBER 21, 2020

UBX1325 is developed from BM-962, a Bcl-xL inhibiting compound licensed to UNITY by Ascentage Pharma for the treatment of age-related diseases. UNITY is a biotechnology company developing therapeutics to extend healthspan by slowing, halting or reversing diseases of aging. SUZHOU, China and ROCKVILLE, Md. ,

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Broad Institute

JULY 10, 2023

Credit: Allison Colorado, Broad Communications Arriving in the Bay Area after a childhood in increasingly diverse surroundings, Martin became acutely aware of health disparities and the profound differences in disease prevalence among different populations. I wanted to know how I could have any impact on studying genetic diversity.”

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Codon

JANUARY 21, 2024

Today, refined versions of these human challenge studies have become standard practice in testing vaccines for vector-borne diseases (e.g., yellow fever, malaria, and dengue), evaluating new drugs or treatments, and studying pathogenesis, the process by which a disease develops.

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The Pharma Data

AUGUST 19, 2021

The Janssen Pharmaceutical Companies of Johnson & Johnson today announced preliminary data from the Phase 1 CHRYSALIS study evaluating RYBREVANT TM (amivantamab-vmjw) for the treatment of patients with non-small cell lung cancer (NSCLC) with mesenchymal-epithelial transition (MET) exon 14 skipping (METex14) mutations. 4] , [5] , [6].

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The Pharma Data

SEPTEMBER 9, 2020

CERENOVUS Launches New Suite of Technologies to Advance Stroke Treatment. CERENOVUS Launches New Suite of Technologies to Advance Stroke Treatment. CERENOVUS offers a broad portfolio of devices used in the endovascular treatment of hemorrhagic and ischemic stroke. Stroke Research and Treatment, Hindawi, 27 Nov.

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New Drug Approvals

SEPTEMBER 23, 2024

Target: DNA/RNA Synthesis Pathway: Cell Cycle/DNA Damage Huntington’s disease (HD) is a progressive, autosomal dominant neurodegenerative disorder of the brain, having symptoms characterized by involuntary movements, cognitive impairment, and mental deterioration. SCHEME PATENT PTC Therapeutics Inc., Audiol Res. 2020-01-02. 2020-01-02.

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MAY 5, 2022

Validated Measurable Residual Disease Assays are Critical for the Rapid Development of Future Novel Therapeutics for Patients with Acute Myeloid Leukemia. At Janssen, we’re creating a future where disease is a thing of the past. About Acute Myeloid Leukemia. About the Janssen Pharmaceutical Companies of Johnson & Johnson.

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The Pharma Data

JULY 15, 2021

The access to the world’s largest browsable resource linking rare protein-coding genetic variants to human health and disease was launched through a genetic exome sequence analysis collaboration between AbbVie (NYSE: ABBV), Biogen Inc. Nasdaq: BIIB) and Pfizer (NYSE: PFE). The browser can be accessed via [link]. About AbbVie.

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The Pharma Data

MARCH 16, 2022

The Independent Data Monitoring Committee recommended that the EMPA-KIDNEY trial be stopped early, following a formal interim assessment EMPA-KIDNEY is the largest and broadest dedicated SGLT2 inhibitor trial in chronic kidney disease to date Detailed results are expected to be presented later this year.

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The Pharma Data

MARCH 30, 2021

– Migraine is a debilitating neurological disease affecting 39 million people in the U.S.[2] – Migraine is a debilitating neurological disease affecting 39 million people in the U.S.[2] AbbVie anticipates a regulatory decision in late Q3 2021. 3 Migraine symptoms and severity range widely among individuals.

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