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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The Pharma Data

OCTOBER 30, 2020

Dive into this week’s update for more details on the actions taken by the FDA in the ongoing response to the Covid-19 pandemic. FDA approves first treatment for Covid-19. On October 22, the FDA approved the antiviral drug Veklury for use in adult and pediatric patients for the treatment of Covid-19 requiring hospitalization.

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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. 15, 2020 (GLOBE NEWSWIRE) — Athenex, Inc., 15, 2020 (GLOBE NEWSWIRE) — Athenex, Inc., The FDA approval of Klisyri is a significant milestone for Athenex. BUFFALO, N.Y., BUFFALO, N.Y.,

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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

DECEMBER 16, 2020

17, 2020 /PRNewswire/ — CNS Pharmaceuticals, Inc. The Company will initiate its trial during the first quarter of 2021 to investigate the efficacy of Berubicin in adults with GBM who have failed first-line therapy. and 2 trials planned by our sublicensee WPD in Poland. . . HOUSTON , Dec.

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The Pharma Data

DECEMBER 23, 2020

On December 3, the PureTech initiated a global, Phase II trial of LYT-100 (deupirfenidone) in Long COVID respiratory complications and related sequelae. The trial is ongoing in the U.S. Leronlimab is currently undergoing a Phase IIb/III trial to evaluate its efficacy and safety for patients with severe-to-critical COVID-19.

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The Pharma Data

SEPTEMBER 8, 2020

Phase III clinical trial programme initiated for investigational medicine fenebrutinib, designed to be a highly selective and reversible Bruton’s tyrosine kinase (BTK) inhibitor, in relapsing multiple sclerosis (RMS) and primary progressive MS (PPMS). Roche’s Chief Medical Officer and Head of Global Product Development. “In

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The Pharma Data

DECEMBER 16, 2020

17, 2020 /PRNewswire/ — The U.S. Food and Drug Administration (FDA) has approved Benlysta to treat lupus nephritis (lupus-related kidney disease) in adults. The approval is for both the intravenous and subcutaneous formulations. WASHINGTON , Dec. Department of Defense Medical Research Program.

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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

MAY 7, 2021

under an Emergency Use Authorization (EUA) granted by the FDA on December 11, 2020. Submission of a BLA, which requires longer-term follow-up data for acceptance and approval, is the next step in the rigorous FDA review process. “We The Pfizer-BioNTech COVID-19 Vaccine is currently available in the U.S.

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The Pharma Data

FEBRUARY 4, 2021

3, 2021 /PRNewswire/ — AbbVie (NYSE: ABBV) announced financial results for the fourth quarter and full year ended December 31, 2020. In both Phase 3 trials, significantly more patients treated with Skyrizi achieved the primary endpoint of ACR20 response at week 24 versus placebo. NORTH CHICAGO, Ill., Recorded a $4.7

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The Pharma Data

APRIL 7, 2022

This unprecedented CMS decision effectively denies all Medicare beneficiaries access to ADUHELM ® (aducanumab-avwa), the first and only FDA-approved therapy in a new class of Alzheimer’s drugs. It may also limit coverage for any future approved treatment in the class.

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New Drug Approvals

DECEMBER 19, 2022

2020 Feb 27;63(4):1612-1623. Epub 2020 Feb 12. 1] Olutasidenib was approved for medical use in the United States in December 2022. [1] 1] Olutasidenib was approved for medical use in the United States in December 2022. [1] Olutasidenib FT-2102 FT2102 C 18 H 15 ClN 4 O 2 354.79 J Med Chem. doi: 10.1021/acs.jmedchem.9b01423.

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The Pharma Data

DECEMBER 13, 2020

Food and Drug Administration (FDA) on Friday, December 11, 2020. During the meeting, the FDA provided encouraging feedback regarding the Phase 3 study of omidubicel pertaining to the pre-specified primary and secondary endpoints. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

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The Pharma Data

JANUARY 6, 2021

The announcement, following a $98 million IPO in October 2020, comes as the company advances several programs through its pipeline. In addition to its lead program, which began Phase I testing in 2020, the company expects to nominate three clinical candidates in 2021.

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New Drug Approvals

SEPTEMBER 8, 2024

1] Palopegteriparatide was approved for medical use in the European Union in November 2023, [2] and in the United States in August 2024. [1] 5] History The effectiveness of palopegteriparatide was evaluated in a 26-week, randomized, double-blind, placebo-controlled trial that enrolled 82 adults with hypoparathyroidism. [5]

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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. Food and Drug Administration (FDA) approval. The FDA Since 1983.

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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. Topline results from the BRIDGE study were announced in May 2020.

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The Pharma Data

MAY 17, 2021

The authority to change drug labels outside of considerations for new safety information “could encourage third parties, such as academic investigators, insurance companies, and cooperative trial groups, to initiate such changes,” they wrote. . The median exclusivity of UDI-approved drugs was 3.42

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The Pharma Data

NOVEMBER 3, 2020

4, 2020 13:00 UTC. DEXTENZA is FDA approved for the treatment of ocular inflammation and pain following ophthalmic surgery. The safety of DEXTENZA was assessed in three Phase 3 clinical trials and a Phase 2 clinical trial. BEDFORD, Mass.–( –( BUSINESS WIRE )– Ocular Therapeutix , Inc.

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The Pharma Data

MARCH 11, 2021

Retevmo was approved under the FDA’s Accelerated Approval regulations based on the LIBRETTO-001 Phase 1/2 trial’s endpoints of overall response rate (ORR) and duration of response (DoR). LY3484356, an oral SERD, is currently being studied in a Phase 1/2 clinical trial. Pipeline Highlights.

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The Pharma Data

NOVEMBER 25, 2020

November 25, 2020 — Under FDA law, approval of a new drug requires substantial evidence of effectiveness and a demonstration of safety for the drug’s intended use(s). The approval of remdesivir ( Veklury ) for the treatment of patients hospitalized with COVID-19 met this legal and scientific standard.

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The Pharma Data

NOVEMBER 9, 2020

09, 2020 (GLOBE NEWSWIRE) — Novavax, Inc. Food and Drug Administration (FDA) has granted Fast Track Designation for NVX-CoV2373, the Company’s COVID-19 vaccine candidate. Novavax expects to begin its pivotal Phase 3 clinical trial in the United States and Mexico by the end of November. GAITHERSBURG, Md., and globally.”.

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The Pharma Data

DECEMBER 30, 2020

30 December 2020 — AstraZeneca’s COVID-19 vaccine has been approved for emergency supply in the UK, with the first doses being released today so that vaccinations may begin early in the New Year. The data were also published in The Lancet on 8 December 2020.

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The Pharma Data

NOVEMBER 30, 2020

30, 2020 (GLOBE NEWSWIRE) — Novavax, Inc. Two of the three planned late-stage efficacy trials for NVX-CoV2373 sponsored by Novavax are fully enrolled, and more than 20,000 participants have been dosed to-date. pivotal Phase 3 trial update. South Africa Phase 2b trial update. Mexico pivotal Phase 3 trial update.

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The Pharma Data

JANUARY 13, 2021

I am pleased with the clinical progress we are making with this program and we are planning to present interim data from the ongoing Phase 1/2 dose escalation trial later this year.”. About the Phase 1/2 Trial for HPN217. Dose escalation for HPN217 in the Phase 1/2 clinical trial is progressing rapidly.

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The Pharma Data

JANUARY 5, 2021

RAD011 is a pivotal-trial ready synthetic cannabidiol oral solution with potential utilization in multiple endocrine and metabolic orphan diseases. Prader-Willi syndrome (“PWS”) will be the initial indication, which has been granted Orphan Drug and Fast Track Designation by the FDA. Disease Highlights.

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The Pharma Data

OCTOBER 25, 2020

26, 2020 (GLOBE NEWSWIRE) — BioXcel Therapeutics, Inc. (“BTI” The Company plans to initiate a Phase 2 trial within the next several months. “We An exploratory endpoint of this trial will be to determine the overall clinical improvement after drug administration using the Clinical Global Impression – Improvement Scale (“CGI-I”).

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The Pharma Data

SEPTEMBER 7, 2020

Novartis will present 48 abstracts from its leading MS portfolio, including new data on recently FDA-approved Kesimpta ® (ofatumumab) — the first and only self-administered, targeted B-cell therapy for relapsing forms of MS (RMS )— Mayzent ® (siponimod) and Gilenya ® (fingolimod) .

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The Pharma Data

NOVEMBER 23, 2020

23 November 2020 . Positive high-level results from an interim analysis of clinical trials of AZD1222 in the UK and Brazil showed the vaccine was highly effective in preventing COVID-19, the primary endpoint, and no hospitalisations or severe cases of the disease were reported in participants receiving the vaccine.

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The Pharma Data

MAY 27, 2022

The FDA approves around 50 new drugs each year after clinical trials and data analyses demonstrate a drug’s safety and effectiveness. To expedite getting potentially beneficial new drugs to seriously ill patients, some of these new drugs are approved through the FDA’s accelerated approval pathway.

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The Pharma Data

NOVEMBER 23, 2020

The study is an open-label, exploratory phase I/II trial adding ONCOS-102 to SoC chemotherapy (pemetrexed/cisplatin) in first- and second- (and later) line treatment of MPM to assess safety, immune activation and efficacy versus SoC only. months (Baas 2020), and this is expected to serve as a benchmark for further approvals.

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The Pharma Data

DECEMBER 28, 2020

29, 2020 /PRNewswire/ — Moleculin Biotech , Inc., . HOUSTON , Dec. About Moleculin Biotech, Inc. Moleculin Biotech, Inc.

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New Drug Approvals

JANUARY 3, 2023

Adagrasib Formula C32H35ClFN7O2 cas 2326521-71-3 Mol weight 604.1174 Antineoplastic Disease Non-small cell lung cancer 2022/12/12 FDA APPROVED, KRAZATI (Mirati Therapeutics) MRTX-849 MRTX849 KRAS G12C inhibitor MRTX849 Adagrasib , sold under the brand name Krazati , is an anticancer medication used to treat non-small cell lung cancer. [1]

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The Pharma Data

DECEMBER 10, 2020

11, 2020 02:48 UTC. Pending discussions with the FDA, the oral vaccine will enter Phase I trials as a prime and boost, and will be explored to provide a boost to subcutaneous vaccinations. For more information about ImmunityBio’s COVID-19 vaccine trials, please contact clinicalresearch@hoag.org. CULVER CITY, Calif.–(

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The Pharma Data

OCTOBER 21, 2020

21 October 2020 — ViiV Healthcare, the global specialist HIV company majority owned by GSK, with Pfizer Inc. These findings were presented today at the 2020 Infectious Diseases Society of America (IDSA) IDWeek. Posted: October 2020. Related Articles: Cabenuva (cabotegravir and rilpivirine) FDA Approval History.

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FDA Law Blog: Biosimilars

FEBRUARY 29, 2024

The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval. If the confirmatory trial shows that the drug actually provides a clinical benefit, then the FDA grants traditional approval for the drug. 9,273,141 was submitted on September 18, 2020 (Docket No.

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