FDA Law Blog: Drug Discovery

FEBRUARY 2, 2023

The 2017 Predictive Toxicology Roadmap laid out some of the FDA’s thinking around the need for new toxicology methods driven in part by a desire to find alternatives to animal testing. 42 U.S.C. § 262(k)(2)(A)(i)(I). However, there are signs that FDA is receptive from a policy perspective to alternative methods.

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The Pharma Data

MARCH 22, 2021

In December 2017, Roche licensed the investigational molecule from Ionis Pharmaceuticals. Tominersen, previously IONIS-HTTRx or RG6042, is an investigational antisense therapy designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT.

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Agency IQ

AUGUST 16, 2024

Throughout 2017, Lykos sought feedback on the protocol for its first Phase 3 study, MAPP1, via the Special Protocol Assessment (SPA) process. Following some back-and-forth regarding statistical analyses and endpoints and abuse potential studies, the agency agreed to a revised SPA in July 2017.

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The Pharma Data

JANUARY 3, 2021

The Phase 1/2 clinical trial will primarily investigate the safety and tolerability of INZ-701 and characterize its pharmacokinetic and pharmacodynamic profile, including plasma pyrophosphate (PPi) and other biomarker levels, to establish a recommended dosing regimen for further clinical development. Demetrios Braddock, M.D.,

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Agency IQ

SEPTEMBER 15, 2023

In 2017, the FDA expanded the restrictions , which include behind-the-counter sale requiring photo identification with a per-month individual purchase quota, to products containing ephedrine and phenylpropanolamine — effectively narrowing the decongestant drugs approved for over-the-counter sale.

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New Drug Approvals

JANUARY 31, 2024

citation needed ] The pharmacokinetic properties of obeldesivir and improved was first published by Chinese researchers in May 2022. 2017, 60, 1648-1661) in tetrahydrofuran (THF) was added N,N-dimethylaminopyridine (DMAP) (0.03 The Chinese group pursued investigation of obeldesivir independently from Gilead Sciences. mmol) (Siegel et.

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The Pharma Data

JANUARY 3, 2021

The pharmacokinetics of nasally administered Foralumab will also be evaluated. . The primary endpoint of this study was safety of the treatment, and secondary endpoints were to evaluate the effect of treatment on disease severity symptoms, nasal tolerance, sense of smell, and biomarkers for disease progression.

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New Drug Approvals

SEPTEMBER 13, 2024

14] Pharmacokinetics The oral bioavailability of aticaprant is 25%. [1] 21] In August 2017, it was announced that Cerecor had sold its rights to aticaprant to Janssen Pharmaceuticals. [22] 22] [21] Janssen was also experimenting with esketamine for the treatment of depression as of 2017. [21] Dhir A (January 2017).

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The Pharma Data

MARCH 16, 2021

for the treatment of adults with moderate to severe plaque PsO in July 2017 and in July 2020 for adults with active PsA. Efficacy, safety, pharmacokinetic, immunogenicity and biomarker evaluations were performed in the study on a defined schedule. TREMFYA was approved in the U.S. About DISCOVER-2 (NCT03158285) 14.

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The Pharma Data

NOVEMBER 6, 2020

Since October 2017 Biogen and Eisai have collaborated on the development and commercialization of aducanumab globally. Secondary outcomes were: (1) the effect of aducanumab on brain amyloid plaque content as measured by [18F]-florbetapir PET, (2) the pharmacokinetics of aducanumab and (3) the immunogenicity of aducanumab.

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The Pharma Data

DECEMBER 20, 2020

Pending Health Canada’s approval, the Phase 1 trial is designed to test the safety, tolerability and pharmacokinetics of ALS-4 in healthy volunteers. The secondary objective is to assess the pharmacokinetic profile of SAD and MAD of ALS-4 administered orally to healthy subjects. About ALS-4.

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Metabolite Tales Blog

JANUARY 23, 2024

Pharmacokinetics and Disposition of Momelotinib Revealed a Disproportionate Human Metabolite—Resolution for Clinical Development. Pharmacokinetics and Metabolism of Nirmatrelvir. [link] [10] FDA prescribing information for gepirone. link] [11] Zheng et al., Blood 129(13): 1823-1830. link] [13] Eng et al., link] [16] Sanga et al.,

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New Drug Approvals

SEPTEMBER 29, 2024

2017 ; Ilievski et al., The pharmacokinetic profiles of COR388 in AD and controls were reported to be similar. Epub 2017 Nov 6 PubMed. In preclinical work from other labs, infection with P. 2018 ; Ding et al., For a review of the preclinical literature, see Costa et al., In human neurons grown in culture, P. NPJ Aging Mech Dis.

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The Pharma Data

JANUARY 19, 2021

The secondary objective is to assess the pharmacokinetic profile of SAD and MAD of ALS-4 administered orally to healthy subjects. This announcement is not a prospectus within the meaning of the Regulation (EU) n°2017/1129 of 14 June 2017 as amended by Regulations Delegated (EU) n°2019/980 of 14 March 2019 and n°2019/979 of 14 March 2019.

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The Pharma Data

OCTOBER 27, 2020

Medtronic), the premarket approval application (PMA) for the ISR was approved by the FDA in December 2017. In October 2020 , we completed a second clinical study in healthy volunteers to compare the pharmacokinetics of Treprostinil Technosphere to Tyvaso. Implantable System for Remodulin (ISR).

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Drug Target Review

SEPTEMBER 19, 2023

The organoid technology recreates different disease scenarios in addition to the healthy models with age specific phenotypes and enables recreating drug specific Pharmacokinetics that is more relevant to human in vivo system.

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Metabolite Tales Blog

DECEMBER 13, 2023

8(2):3640-3648; [link] [11] Pharmacokinetics of the Multi-kinase Inhibitor Pexidartinib: Mass Balance and Dose Proportionality. J Pharm Biomed Anal. 22(5):803-11; [link] [10] Glucuronidation and UGT isozymes in bladder: new targets for the treatment of uroepithelial carcinomas? Sundararaghavan VL, Sindhwani P, Hinds TD Jr. Oncotarget.

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Alta Sciences

APRIL 17, 2024

The FDA approved the first gene therapy in 2017 for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL), and as of December 2023, there are over 30 approved cell and gene therapies on the market in the U.S.A.

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The Pharma Data

NOVEMBER 9, 2020

The pharmacokinetic tail—will this be a significant risk for drug resistance? It also has a long pharmacokinetic. It is not yet understood if this long pharmacokinetic tail will have any significant effect on drug resistance. iv WHO, 2017 WHO implementation tool for pre-exposure prophylaxis of HIV infection.

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Metabolite Tales Blog

MARCH 1, 2023

2017, 30, 1, 13–37 [link] 2a Drug Metab. 2010, 75, 9, 3141–3143 [link] 22 Clin Pharmacokinet. There is much to be learnt from the pathways described above but in general, the more nitrogens present in your ring, the less likely you are to see Phase I type oxidations of the ring itself. Instead, be on the lookout for N -glucuronides.

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Drug Target Review

OCTOBER 4, 2023

7 These inhibitors have faced challenges such as dose-limiting toxicity and poor pharmacokinetics, but geldanamycin ADCs have demonstrated increased survival in mice. 56 , 733–737 (2017). 9 Others, like psymberin, have been used as ADC payloads, with a β-glucuronide linker to target CD-30-positive and CD-70-positive malignancies.

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The Pharma Data

AUGUST 22, 2021

Safety, Tolerability, and pharmacokinetics of MEDI8897, the respiratory syncytial virus prefusion F-targeting monoclonal antibody with an extended half-life, in healthy adults. 2017; 61(3): e01714-16. Griffin MP, et al. Antimicrob Agents Chemother. Yu XQ, et al. Antimicrob Agents Chemother. 2016; 61 (1): e01020-16.

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Agency IQ

FEBRUARY 23, 2024

This workshop built on three prior workshops held by the FDA-AACR, which took place in 2015, 2016 and 2017. And I do think it’s really important to go as high up as we can safely, because there are issues that have already been mentioned – inter-tumor heterogeneity, issues of tumor penetration and [pharmacokinetics] PK.”

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Agency IQ

AUGUST 16, 2024

What came before this final guidance Dose optimization for oncology products has been an issue flagged for improvement even prior to the creation of FDA’s Oncology Center of Excellence (OCE) in 2017. In parallel, stakeholders have advocated for recognizing the importance of treatment tolerability when determining doses and regimens.

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The Pharma Data

DECEMBER 15, 2020

The Phase I/IIa clinical trial is a randomized, double-blind, placebo controlled, single and multiple dose escalation study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of NMD670 in 79 male and female healthy subjects and patients with myasthenia gravis. The secondary outcome involves pharmacokinetic endpoints.

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The Pharma Data

APRIL 8, 2021

EVRYSDI was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by FDA and EMA in 2017 and 2019, respectively. Food and Drug Administration (FDA) approved EVRYSDI for the treatment of SMA in adults and children 2 months of age and older. About OCREVUS ® (ocrelizumab).

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The Pharma Data

FEBRUARY 8, 2021

Additionally, ZYTIGA ® was approved for the treatment of high-risk mCSPC by the European Commission on November 20, 2017 and by the U.S. In a dedicated drug interaction trial, co-administration of ketoconazole, a strong inhibitor of CYP3A4, had no clinically meaningful effect on the pharmacokinetics of abiraterone.

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The Pharma Data

SEPTEMBER 15, 2020

Of note, The Phase 1b study is a double-blind, placebo-controlled clinical trial evaluating the safety, tolerability and pharmacokinetics of PF-07304814, a phosphate prodrug that when administered intravenously is metabolized to the active compound PF-00835321, shown to be a very potent inhibitor of the SARS-Cov2 3CL protease in preclinical studies.

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Agency IQ

DECEMBER 11, 2023

Oncology at FDA in 2023: A quick glance at the numbers First created under the 21st Century Cures Act of 2016 and launched in 2017, FDA’s Oncology Center of Excellence (OCE ) “unites experts across the FDA to conduct expedited review of medical products for oncologic and hematologic malignancies.”

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Agency IQ

FEBRUARY 15, 2024

CDRH FY2024 Guidance Agenda Clinical application Recommendations for Investigational and Licensed COVID-19 Convalescent Plasma CBER Final By 12/31/24 CBER CY 2024 Guidance Agenda Blood Considerations for the Development of Blood Collection, Processing, and Storage Systems for the Manufacture of Blood Components Using the Buffy Coat Method CBER Draft (..)

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The Premier Consulting Blog

APRIL 25, 2023

In silico population pharmacokinetic (popPK) and physiologically-based pharmacokinetic (PBPK) modeling can reduce or eliminate certain clinical studies, as was done with the 505(b)(2) approval of the long-acting injectable Aristada® (aripiprazole lauroxil). [8] ICH Harmonised Guideline S10: Photosafety evaluation of pharmaceuticals.

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The Pharma Data

OCTOBER 6, 2021

Safety, tolerability, and pharmacokinetics of MEDI8897, the respiratory syncytial elixir prefusion F-targeting monoclonal antibody with an extended half- life, in healthy grown-ups. 2017; 61 (3) e01714-16. Griffin MP, et al. Antimicrob Agents Chemother. Yu XQ, et al. Antimicrob Agents Chemother. 2016; 61 (1) e01020-16.

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The Pharma Data

FEBRUARY 25, 2021

FIREFISH, an open-label, two-part pivotal study, was designed to assess Evrysdi safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) in patients aged 1 to 7 months with Type 1 SMA. Part 1 evaluated several doses of Evrysdi and determined the therapeutic dose of 0.2 mg/kg for Part 2.

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The Pharma Data

MARCH 20, 2023

Food and Drug Administration in 2017. JEWELFISH (NCT03032172) – an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi.

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The Pharma Data

MARCH 16, 2021

Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by FDA and EMA in 2017 and 2019, respectively. At this time, Evrysdi has been approved in seven countries and submitted in 57, including the EU 27 and Norway and Iceland. The study met its primary endpoint.

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The Pharma Data

SEPTEMBER 27, 2020

Risdiplam was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by FDA and EMA in 2017 and 2019, respectively. The FDA approved Evrysdi for the treatment of SMA in adults and children 2 months of age and older. The study met its primary endpoint. The study has completed recruitment (n=174).

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