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. In contrast , the two previous withdrawals, Makena in 2023 and Avastin in 2011, took 30 months and 11 months, respectively.

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Agency IQ

APRIL 12, 2024

Draft guidance on potency assays for CGT products garners extensive stakeholder input Late last year, the FDA published a draft update to its 2011 guidance on potency assays for cell and gene therapy products, unveiling a major shift in approach to the issue. How does “potency” apply to cell and gene therapy (CGT) products?

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The Pharma Data

NOVEMBER 23, 2020

The authors used longitudinal data on 10,676 Medicare-enrolled participants aged 65 years and older from the National Health and Aging Trends Study 2011 to 2018 cycles to assess the likelihood of developing functional visual impairment over time given baseline dementia, and vice versa. ” Abstract No. Abbott, M.D., © 2020 HealthDay.

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New Drug Approvals

SEPTEMBER 13, 2024

2] As of July 2022, it is in phase 3 clinical trials for major depressive disorder. [2] hours following a single dose of 10 mg, which supported the 4 mg to 25 mg dosages that aticaprant is being explored at in clinical trials. [13] 2] It first appeared in the scientific literature in 2010 or 2011. [16] nM vs. 24.0 nM vs. 24.0

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The Pharma Data

AUGUST 9, 2020

Etrolizumab failed to meet its primary endpoint versus placebo as maintenance therapy in people with ulcerative colitis. The safety profile of etrolizumab was consistent with previous studies and no major safety issues were identified in the four phase III clinical trials reported to date. “We

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The Pharma Data

DECEMBER 16, 2020

The decision makes Benlysta the first lupus therapy authorized to treat this potentially life-threatening complication of the disease. The FDA first approved Benlysta for lupus in 2011. Up to 60 percent of people with lupus will develop lupus nephritis. Benlysta is a human monoclonal antibody sold by GSK.

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Advarra

NOVEMBER 10, 2022

multisite research and trials). Many industry sponsors already request participating sites in multisite trials rely on the study’s central IRB as a way to reduce the administrative burden of managing multiple IRB requirements, processes, and timelines, citing FDA’s 2006 guidance on centralized IRB review.

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The Premier Consulting Blog

NOVEMBER 20, 2024

Stages of process validation Process validation involves three distinct stages as described in the 2011 FDA guidance, “Process Validation: General, Principles and Practices.” The quality of data from Stage I validation activities. Prior PPQ knowledge and experience with similar products.

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The Pharma Data

NOVEMBER 15, 2020

This prestigious group of professionals will help lead the Company as it prepares for potential commercialization of its investigational cell therapy, MultiStem ®. Prior to Celgene, she held leadership roles in marketing, sales and new business development from 2002 to 2011 at Johnson & Johnson. Before that, Ms. About MultiStem ®.

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The Pharma Data

NOVEMBER 5, 2020

Velders is presently chairman of HollandBIO, the association of Dutch biotech companies, and provides years of experience, specifically in the cell and gene therapy areas. Taveras will lead all research and non-clinical development functions supporting the company’s pipeline of investigational therapies.

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FDA Law Blog: Biosimilars

JULY 24, 2023

And, because comparative effectiveness claims must be supported by substantial evidence—one or more adequate and well-controlled clinical trials—FDA’s decision cannot stand as Avadel never conducted a comparative clinical trial of Xywav and Lumryz. Thus, Jazz argues, there is no evidence that Lumryz is more effective than Xywav.

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Agency IQ

SEPTEMBER 15, 2023

The clinical trials underpinning the original determination that phenylephrine is GRASE – that is, Generally Regarded as Safe and Effective – predate the 1994 inclusion of phenylephrine hydrochloride in the final monograph for OTC nasal decongestant drug products. Both trials showed no difference between phenylephrine and placebo.

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PLOS: DNA Science

MARCH 21, 2024

The Food and Drug Administration just announced approval of Lenmeldy (atidarsagene autotemcel), a gene therapy to treat the neurological condition metachromatic leukodystrophy (MLD). 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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Broad Institute

OCTOBER 11, 2023

By Allessandra DiCorato October 11, 2023 In 2011, Robert Manguso was working in a cell biology lab when his mother was diagnosed with Merkel cell carcinoma, a rare and aggressive skin cancer. By then, a few cancer immunotherapies had entered clinical trials and only a handful had been approved by the FDA.

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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. As such, it doesn’t review things like vaccines, blood products or gene therapies – those products are instead reviewed by CBER.

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Drug Target Review

JUNE 21, 2023

Enabling the systematic discovery of these largely uncharted targets can be a valuable opportunity for development of novel therapies. Genetic-based therapeutics hold significant promise in the clinic Despite the growing number of genes linked to human disease, the proportion of genes targeted by approved therapies remains low.

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The Pharma Data

DECEMBER 17, 2020

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. First approved in 2011, belimumab is now the first and only biologic approved for both SLE and LN in over a half-century. MacroGenics Inc. Genentech Inc.

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Codon

APRIL 9, 2024

After confirming they experienced no side effects, Tu and her colleagues carried out the first clinical trial on human patients in Hainan between August and October 1972, which continued to demonstrate qinghao extract’s high efficacy. Miller and Su published an article on Tu’s story in Cell in 2011.

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The Pharma Data

AUGUST 11, 2020

.–( BUSINESS WIRE )– Bristol Myers Squibb (NYSE: BMY) today announced the Phase 3 CheckMate -577 trial evaluating Opdivo (nivolumab) as an adjuvant therapy for patients with resected esophageal or gastroesophageal junction (GEJ) cancer met its primary endpoint of disease-free survival (DFS) at a pre-specified interim analysis.

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The Pharma Data

JANUARY 6, 2021

The REVIVAL Phase III pivotal trial is a randomized, double-blind, placebo-controlled, two-arm, parallel-group, multi-center trial to evaluate the efficacy and safety of AM-Pharma’s proprietary human recombinant alkaline phosphatase for the treatment of patients with SA-AKI. Professor Peter Pickkers, M.D., billion on an annual basis.

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The Pharma Data

JANUARY 20, 2021

The combination therapy involves an injectable medication called extended-release naltrexone and a daily generic pill called bupropion. But between 2017 and 2019, more than 400 adults with moderate-to-severe methamphetamine use disorder participated in a combination treatment trial, and the results suggest the combination works.

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The Pharma Data

AUGUST 11, 2020

The safety profiles of Opdivo and chemotherapy in this trial are reflective of the known safety profiles of Opdivo and chemotherapy in first-line gastric and esophageal cancers. The company remains blinded to data from this arm and the trial continues in follow-up to allow the data to mature. PRINCETON, N.J.–(

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The Pharma Data

NOVEMBER 10, 2020

While the world is facing a severe shortage of specific antiviral drugs, a new treatment developed by Asclepius Meditec highlights the potential of hydrogen-oxygen therapy in tackling COVID-19. Founded in 2011, Shanghai Asclepius Meditec is the first company globally to launch clinical research on hydrogen-oxygen medicine.

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Agency IQ

SEPTEMBER 22, 2023

Traditionally, FDA has interpreted the need for “well-controlled investigations” to mean at least two clinical trials for new drugs, or applications for supplemental indications. When looking at all drugs approved between 2011 and May 2020, 136 drugs were approved on the basis of a single pivotal trial, according to AgencyIQ’s research.

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The Pharma Data

DECEMBER 3, 2020

An initial analysis of data from 17/22 patients who completed one of these assessments showed substantially decreased neurocognitive abilities compared to children of the same age level despite the use of multiple anti-epileptic therapies. Clinical Trials. December 6, 2020 12:00 PM – 1:30 PM; Track: 7. Antiepileptic Drugs / 7B.

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The Pharma Data

SEPTEMBER 8, 2021

CheckMate -743 remains the only Phase 3 trial to show a survival benefit with first-line immunotherapy treatment in patients with MPM. First-line nivolumab + cabozantinib vs sunitinib in patients (pts) with advanced renal cell carcinoma (aRCC) in subgroups based on prior nephrectomy in the CheckMate 9ER trial. Summary of Presentations.

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The Pharma Data

MAY 5, 2021

Farxiga received fast track, breakthrough therapy and priority review designations for the current indication. . Ferriprox was originally approved in 2011 as a therapy for patients with transfusional iron overload due to thalassemia syndromes for whom chelation therapy was not adequate. . blind, placebo?controlled

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The Pharma Data

DECEMBER 21, 2020

DFS results in the overall trial population of patients with Stage IB-IIIA disease showed Tagrisso reduced the risk of disease recurrence or death by 80% (HR 0.20; 95% CI 0.15-0.27; The safety and tolerability of Tagrisso in this trial was consistent with previous trials in the metastatic setting. 0.23; p<0.0001).

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Agency IQ

OCTOBER 6, 2023

As such, it doesn’t review things like vaccines, blood products or gene therapies – those products are instead reviewed by CBER. This was more than twice the volume seen in FY 2022 and the second highest percentage of novel drug approvals in a given year dating back to 2011. of all NME approvals this year.

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The Pharma Data

JUNE 26, 2021

Current guidelines are limited and recommend treating pediatric patients with or at risk for reoccurring blood clots with standard anticoagulant therapy, which often requires painful injections, dietary restrictions and regular laboratory monitoring. EINSTEIN-Jr is the largest pediatric study completed to date for the treatment of VTE.

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The Pharma Data

FEBRUARY 25, 2022

Jardiance is the first and only heart failure therapy to demonstrate a statistically significant risk reduction in cardiovascular death or hospitalization for heart failure, regardless of ejection fraction FDA approval marks a significant breakthrough for the approximately 3 million adults in the U.S. and Europe.

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The Pharma Data

DECEMBER 13, 2020

About 15 to 20% of breast cancers cases are HER2+, and antibody-based therapies targeting HER2 have significantly improved outcomes of patients with HER2+ breast cancer. The biosimilar is a collaboration between Amgen and Allergan, which was formed when Allergan was Watson Pharmaceuticals in December 2011.

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The Pharma Data

AUGUST 3, 2021

In 2011, Celgene Corporation, now a wholly owned subsidiary of Bristol Myers Squibb, received accelerated approval by the U.S. Istodax remains on the market for treatment of patients with cutaneous T-cell lymphoma (CTCL) who have received at least one prior systemic therapy. About Istodax. Indication. Important Safety Information.

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Codon

JUNE 26, 2023

Since the last Codon Digest, I’ve published: Reasons to Be Grateful for Biotechnology (with Avadhoot Jadhav) AAV Foundations (Part I) An overview of AAV-based gene therapies, how they get made, and where they go wrong. I pulled them from a 2011 blog post ( oH mY goD tHat’S nOt pEEr-ReVieWeD!!! ) Let’s be real.

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Codon

JUNE 26, 2023

Since the last Codon Digest, I’ve published: Reasons to Be Grateful for Biotechnology (with Avadhoot Jadhav) AAV Foundations (Part I) An overview of AAV-based gene therapies, how they get made, and where they go wrong. I pulled them from a 2011 blog post ( oH mY goD tHat’S nOt pEEr-ReVieWeD!!! ) Let’s be real.

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The Pharma Data

OCTOBER 25, 2020

In a sample of charts from 56 patients with SCD, voxelotor increased hemoglobin by more than 1 g/dL on average and decreased hemolysis markers to a degree consistent with the randomized controlled HOPE trial results. . Details of the ASCAT presentations are as follows: Wednesday , Oct. GBT Time: 11 a.m. 4 – 7.

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The Pharma Data

APRIL 28, 2021

1 The submissions were supported by two pivotal Phase 3 studies, KEEPsAKE-1 and KEEPsAKE-2, which evaluated SKYRIZI in adults with active psoriatic arthritis including those who had responded inadequately or were intolerant to biologic therapy and/or non-biologic disease-modifying anti-rheumatic drugs (DMARDs). The Lancet. Reich, K.,

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