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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The Premier Consulting Blog

JUNE 18, 2023

One exciting application of these technologies is the use of in silico trials in the development of novel therapies for rare diseases. However, with rare diseases there may be no available treatments that could serve as SoC or active control in a clinical trial and assigning patients to placebo may be unethical.

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Agency IQ

JULY 22, 2024

Halfway there: Novel drug approvals and their supportive clinical trials so far in 2024 In the first half of 2024, the FDA’s Center for Drug Evaluation and Research (CDER) approved 21 novel drug products. Most NMEs approved so far this year relied on one pivotal trial.

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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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New Drug Approvals

SEPTEMBER 29, 2024

gingivalis has been called a risk factor for Alzheimer’s disease ( Kanagasingam et al., gingivalis is associated with cardiovascular disease. Both were reversed by treatment with COR388 ( 2020 AAIC abstract ). Findings Two Phase 1 trials of atuzaginstat were completed by June 2019. and Europe.

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The Pharma Data

OCTOBER 22, 2020

The randomized, placebo-controlled trial is being conducted in partnership with the NIH’s National Institute of Allergy and Infectious Diseases and HHS’ Biomedical Advanced Research and Development Authority.

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The Pharma Data

OCTOBER 15, 2020

Vertex Pharmaceuticals has decided to give up on its experimental VX-814, a small molecule drug for the rare genetic disease Alpha-1 antitrypsin deficiency (AATD), canning the drug’s development after seeing lackluster results from an early phase 2 trial. James Miessler. Source link.

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Advarra

AUGUST 16, 2022

A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Since then, the FDA has significantly changed its approach to rare and orphan diseases.

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The Pharma Data

NOVEMBER 13, 2020

Centers for Disease Control and Prevention analysis finds. “While health systems are strained by the COVID-19 pandemic, we must not allow our fight against one deadly disease to come at the expense of our fight against another. © 2020 HealthDay. Posted: November 2020. All rights reserved. Source link.

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The Pharma Data

DECEMBER 29, 2020

Phase III BRIDGE open-label, switch-over clinical trial met key objectives for safety and efficacy. 30, 2020 /PRNewswire/ — Protalix BioTherapeutics, Inc. . 30, 2020 /PRNewswire/ — Protalix BioTherapeutics, Inc. galactosidase-A product candidate under development for the treatment of Fabry disease.

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The Pharma Data

OCTOBER 26, 2020

The NIH’s National Institute of Allergy and Infectious Diseases (NIAID) has halted a clinical trial evaluating Eli Lilly’s investigational monoclonal antibody, LY-CoV555 (bamlanivimab), in combination with remdesivir for the treatment of COVID-19 patients. The trial was paused earlier this month due to a safety issue.

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The Pharma Data

OCTOBER 14, 2020

15, 2020 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. Conference Call Information : Date : Thursday, November 5, 2020. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

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Quanticate

MAY 3, 2020

This article has been updated in May 2020 to reflect the recent guidance by European Medicines Agency and other regulatory bodies on remote monitoring due to the recent coronavirus disease (COVID-19) pandemic. With this increase comes the enhanced pressure of effectively monitoring these trials.

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The Pharma Data

JUNE 26, 2021

Sanofi and Translate Bio initiate Phase 1 clinical trial of mRNA influenza vaccine. The trial will evaluate the safety and immunogenicity of a monovalent flu vaccine candidate coding for the hemagglutinin protein of the A/H3N2 strain of the influenza virus. JUNE 22 , 2021.

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The Pharma Data

OCTOBER 26, 2020

27, 2020 /PRNewswire/ — Synlogic, Inc. Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, today announced the Company will release its third quarter 2020 financial results before the market opens on Thursday, November 5, 2020. CAMBRIDGE, Mass. ,

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The Pharma Data

NOVEMBER 29, 2020

AstraZeneca’s COVID-19 vaccine candidate is facing another potential delay as the company plans to undertake a new global clinical trial to confirm the vaccine’s 90 percent efficacy based on a one-and-a-half-dose regimen. An additional trial could further delay AZ’s efforts to earn an EUA in the U.S., James Miessler. Source link.

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The Pharma Data

DECEMBER 8, 2020

8, 2020 — AstraZeneca’s COVID-19 vaccine is safe and effective, new data from late-stage trials shows. Overall, the vaccine protected against symptomatic disease in 70% of cases, according to a team led by researchers from Oxford University in England. TUESDAY, Dec. 8 in The Lancet.

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The Pharma Data

NOVEMBER 12, 2020

12, 2020 — Hydroxychloroquine is not beneficial for adults hospitalized with COVID-19 , according to the formal conclusion of a study funded by the U.S. Patients were randomly assigned to either hydroxychloroquine or placebo (242 and 237 patients, respectively); the trial was stopped for futility at the fourth interim analysis.

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The Pharma Data

AUGUST 22, 2021

The trial accrued 25 cases of symptomatic COVID-19 at the primary analysis.There were no cases of severe COVID-19 or COVID-19-related deaths in those treated with AZD7442. The trial included 5,197 participants in a 2:1 randomisation AZD7442 to placebo. The trial was conducted in 87 sites in the US, UK, Spain, France and Belgium.

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The Pharma Data

OCTOBER 29, 2020

29, 2020– Alexion Pharmaceuticals, Inc. NASDAQ:ALXN) today announced financial results for the third quarter of 2020. Non-GAAP diluted EPS for the third quarter of 2020 was $3.24 , a 16 percent increase versus the third quarter of 2019. ” Third Quarter 2020 Financial Highlights. . in the prior year.

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The Pharma Data

NOVEMBER 16, 2020

November 16, 2020 — An independent data and safety monitoring board (DSMB) overseeing the Phase 3 trial of the investigational COVID-19 vaccine known as mRNA-1273 reviewed trial data and shared its interim analysis with the trial oversight group on Nov. 37% of trial volunteers are from racial and ethnic minorities.

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The Pharma Data

NOVEMBER 18, 2020

18, 2020 — Children should be included in clinical trials for a severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) vaccine at the earliest stages, according to a letter from the president of the American Academy of Pediatrics (AAP), on behalf of more than 67,000 pediatricians and pediatric medical and surgical subspecialists.

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The Pharma Data

APRIL 25, 2021

Nirsevimab demonstrate d protect ion against respiratory syncytial virus disease in healthy infants in Phase 3 trial. The overall safety profile of nirsevimab in the trial remains consistent with previously reported results. Respiratory Syncytial Virus (RSV) is the leading cause of hospitalization in all infants. 1 – 5.

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The Pharma Data

JANUARY 27, 2021

a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, today announces the enrollment of the first participant in Europe in its global Phase 3 clinical trial for HD. The first US subjects in the PROOF-HD trial were enrolled in October 2020.

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The Pharma Data

NOVEMBER 11, 2020

. Received Rare Pediatric Disease and Fast Track Designations for INZ-701 for the treatment of ENPP1 deficiency. Expect to initiate INZ-701 Phase 1/2 clinical trials for ENPP1 and ABCC6 deficiencies in first half of 2021. 12, 2020 (GLOBE NEWSWIRE) — Inozyme Pharma, Inc. BOSTON, Nov.

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The Pharma Data

OCTOBER 28, 2021

Bayer will present new renal and cardiovascular (CV) analyses from the comprehensive finerenone (Kerendia®) clinical trial program, including the Phase III FIGARO-DKD and FIDELIO-DKD studies, and the prespecified pooled analysis FIDELITY at the American Society of Nephrology (ASN)’s Kidney Week 2021 from 4-7 November.

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The Pharma Data

MARCH 16, 2022

(Nasdaq: BIIB) announced that The Journal of Prevention of Alzheimer’s Disease (JPAD) today published a peer-reviewed manuscript detailing data from the pivotal Phase 3 EMERGE and ENGAGE trials for ADUHELM ® (aducanumab-avwa) 100 mg/mL injection for intravenous use in early Alzheimer’s disease.

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The Pharma Data

NOVEMBER 10, 2020

Innovent has established a robust pipeline of 23 valuable assets, including four launched products, four products in pivotal trials, and multiple prioritized assets such as CD47, PDL1/CD47, TIGIT, VEGF/complement protein etc. that are fast progressing clinical trials. SAN FRANCISCO and SUZHOU, China , Nov.

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The Pharma Data

DECEMBER 31, 2020

Monthly News Roundup – December 2020. In studies, the vaccines were found to be 94% to 95% effective in preventing COVID-19 disease. The partial piece of the spike protein cannot cause COVID-19 disease. Posted: December 2020. . Posted: December 2020. In December, the U.S.

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Agency IQ

APRIL 19, 2024

FDA advisors endorse minimal residual disease (MRD) as accelerated approval endpoint for multiple myeloma Last week, FDA’s Oncologic Drugs Advisory Committee (ODAC) voted unanimously in favor of using minimal residual disease (MRD) as an accelerated approval endpoint for multiple myeloma.

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Drug Target Review

NOVEMBER 1, 2023

2 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. 1-5 Implication of ATX in a large range of human diseases have been highlighted by both fundamental research and clinical trials. He is a researcher at University of Oldenburg, Germany.

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The Pharma Data

JUNE 25, 2021

(Nasdaq: NVAX), today announced that NVX-CoV2373, its recombinant nanoparticle protein-based COVID-19 vaccine, demonstrated 100% protection against moderate and severe disease, 90.4% efficacy overall, and met the primary endpoint in its PREVENT-19 pivotal Phase 3 trial. 100) against moderate or severe disease. Government.”

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The Pharma Data

MARCH 11, 2021

Clinical trial to assess safety, immune response and reactogenicity, after preclinical data showed high neutralizing antibody levels. The Companies expect interim results from this trial in the third quarter of 2021. About the Phase 1/2 clinical trial. Expected to enroll 415 participants; interim results expected in Q3 2021.

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The Pharma Data

NOVEMBER 26, 2020

27, 2020 /PRNewswire/ — Protalix Biotherapeutics, Inc. Galactosidase–A protein for the proposed treatment of Fabry disease; OPRX–106, an orally-delivered anti-inflammatory treatment; alidornase alfa for the treatment of Cystic Fibrosis; and others. About Chiesi Global Rare Diseases.

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The Pharma Data

AUGUST 15, 2021

Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.

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The Pharma Data

OCTOBER 27, 2020

Trial of Antibody Drug for COVID-19 Stopped for Lack of Effectiveness. 27, 2020 — Testing of the Eli Lilly antibody drug for hospitalized COVID-19 patients has been halted because the treatment does not help them recover from their infection. © 2020 HealthDay. Posted: October 2020. Professional. TUESDAY, Oct.

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The Pharma Data

DECEMBER 23, 2020

23, 2020 — Because COVID-19 is known to raise the odds for dangerous blood clots, blood thinners have quickly become part of routine care for many hospitalized patients. The three trials are being conducted on four continents. WEDNESDAY, Dec.

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