New Drug Approvals

DECEMBER 24, 2023

2] [3] [4] [5] It was approved for medical use in the United States in December 2023. [6] “Population pharmacokinetic/pharmacodynamic modelling of eplontersen, an antisense oligonucleotide in development for transthyretin amyloidosis” British Journal of Clinical Pharmacology. Food and Drug Administration (FDA). .

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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. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with unique pharmacokinetic parameters. CARMIEL, Israel and BOSTON , Dec. Galactosidase-A enzyme.

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PerkinElmer

JANUARY 7, 2022

There are many reasons that promising drug candidates are discontinued, including poor pharmacokinetics, lack of clinical efficacy, and toxicity. Furthermore, nearly one third of drugs get withdrawn from the market post approval due to safety concerns. link] New safety concerns identified for 1 in 3 FDA-approved drugs [Internet].

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New Drug Approvals

OCTOBER 24, 2023

Molecular Weight: 631.700 FDA APPROVED, To treat moderately to severely active ulcerative colitis in adults, 10/12/2023 Velsipity Etrasimod , sold under the brand name Velsipity , is a medication that is used for the treatment of ulcerative colitis (UC). [1] “FDA Approves New Drug for Ulcerative Colitis” Medscape.

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The Pharma Data

OCTOBER 25, 2020

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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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. We have expertise in both ligand binding and hybrid LC-MS methodologies.

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The Premier Consulting Blog

APRIL 15, 2024

However, when it comes to an IND and supporting a clinical trial, FDA’s primary focus is on healthy volunteer and patient safety. It is critical that the nonclinical program outlined in the PIND briefing document is presented in a manner that allows FDA to provide relevant input on the required IND-enabling studies.

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The Pharma Data

AUGUST 19, 2021

Food and Drug Administration (FDA) approval for patients with locally advanced or metastatic NSCLC with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, based on data showing an ORR of 40 percent (95 percent CI, 29 – 51) and median duration of response of 11.1 months (95 percent CI, 6.9 – NE). [7].

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The Pharma Data

NOVEMBER 26, 2020

Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with unique pharmacokinetic parameters. Protalix was the first company to gain FDA approval of a protein produced through plant cell-based in suspension expression system. About Pegunigalsidase Alfa.

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The Pharma Data

MAY 17, 2023

Additionally, pre-clinical data have shown fenebrutinib to be potent and highly selective, and it is the only reversible inhibitor currently in Phase III trials for MS. “I Fenebrutinib significantly reduced the total number of new gadolinium-enhancing T1 brain lesions compared to placebo, the primary endpoint of the trial (p=0.0022).

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The Pharma Data

JUNE 28, 2021

There are currently no FDA-approved anticoagulation therapies for pediatric patients with congenital heart disease who have undergone the Fontan procedure. EINSTEIN-Jr. About the EINSTEIN-Jr. Study The randomized, open-label phase III EINSTEIN-Jr. UNIVERSE was conducted in two parts.

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The Pharma Data

APRIL 8, 2021

New 2-year findings from Part 2 of the Phase II/III FIREFISH trial show longer-term efficacy and safety of EVRYSDI in infants with symptomatic Type 1 SMA treated with EVRYSDI. P6: Neuromuscular Disorders and Clinical Trials. P6: Neuromuscular Disorders and Clinical Trials. Multiple Sclerosis (MS).

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DrugBank

FEBRUARY 12, 2025

This approach capitalizes on prior investments in R&D, mitigates risk by leveraging established safety and pharmacokinetic profiles, and accelerates the delivery of treatments to patients. The National Institutes of Health (NIH) Clinical Collection , a library of FDA-approved drugs, is widely used for HTS in drug repurposing initiatives.

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The Pharma Data

DECEMBER 15, 2020

SetPoint Medical received FDA Investigational Device Exemption (IDE) approval for a multicenter, double-blind, randomized, sham-controlled pivotal trial that will enroll up to 250 patients at 40 clinical trial sites in the U.S. The secondary outcome involves pharmacokinetic endpoints. Small Molecule Inhibitors.

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The Pharma Data

SEPTEMBER 8, 2021

–Results from a post-hoc analysis of the Phase 3 PREEMPT trials evaluate the use of BOTOX® (onabotulinumtoxinA) for chronic migraine –These data further demonstrate AbbVie’s commitment to harnessing and sharing innovative science and working to advance treatment options for people with migraine across the migraine spectrum.

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The Pharma Data

APRIL 11, 2021

” In the Phase 1/2 LIBRETTO-001 trial, 32 adult patients with 12 unique RET fusion-positive advanced cancer types were enrolled by the efficacy cutoff date of September 19, 2020 (with follow-up through March 19, 2021). Retevmo was approved based on the Phase 1/2 LIBRETTO-001 trial’s endpoints of ORR and DoR.

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Agency IQ

SEPTEMBER 15, 2023

Until recently, the FDA relied on a monograph process through which firms could bring OTC drugs to market without FDA approval so long as it adhered to pre-set terms under the monograph. Finally, the committee came up with a list of recommendations for the structure of additional trials, listed in the meeting minutes.

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The Pharma Data

DECEMBER 5, 2020

Potentially registrational Phase 2 portion of the trial has been initiated and is enrolling patients. NASDAQ: REGN) today announced updated data for REGN5458, a BCMAxCD3 bispecific antibody, from the Phase 1 portion of a Phase 1/2 trial in patients with relapsed or refractory (R/R) multiple myeloma. Regeneron Pharmaceuticals, Inc.

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The Pharma Data

JANUARY 21, 2021

Results from these trials were presented at the 2019 Conference on Retroviruses and Opportunistic Infections (CROI). Hepatic Impairment: EDURANT ® should be used with caution in patients with severe hepatic impairment (Child-Pugh Class C) as pharmacokinetics of EDURANT ® have not been evaluated in these patients.

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The ChEMBL-og

APRIL 16, 2024

The data in MOLECULE_DICTIONARY.MAX_PHASE now includes consideration of: EMA approved drugs (max_phase=4 for human drugs), USAN clinical candidate drugs (assigned as max_phase = 1 based on USAN guidance that states “Firms usually apply for a USAN when the investigational therapy is in Phase I or Phase II trials”.

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The Pharma Data

MAY 5, 2021

It also provided supporting pharmacokinetic data demonstrating the opioid antagonist’s safety and efficacy. . The approval was based on results in a prespecified interim analysis of the first 264 patients of the ongoing KEYNOTE-811 trial. controlled trial had not received previous systemic therapy for metastatic disease.

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The Pharma Data

SEPTEMBER 1, 2021

The FDA approval of INVEGA HAFYERA™ is based on the results of a 12-month, randomized, double-blind, non-inferiority Phase 3 global study that enrolled 702 adults (ages 18-70) living with schizophrenia from 20 countries. DFAPA, Medical Director at ATP Clinical Research and 6-month paliperidone palmitate clinical trial investigator.

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The Pharma Data

APRIL 27, 2021

The NDA for mobocertinib is primarily based on results from the Phase 1/2 trial , which is evaluating the safety and efficacy of oral mobocertinib in patients with mNSCLC. The application was submitted under the FDA’s accelerated approval program. About the Phase 1/2 Trial. and around the globe.”.

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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. Three drugs were approved based on no pivotal trials (such as in cases where an approval leveraged literature on use of the drug).

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The Pharma Data

MARCH 16, 2021

Evrysdi is the first and only at home SMA treatment approved by the FDA, and has proven efficacy across adults, children and infants 2 months and older. More than 2,500 patients now treated with Evrysdi in clinical trial, compassionate use and real-world settings. The study met its primary endpoint.

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Codon

APRIL 2, 2023

Read Safety and pharmacokinetics of escalating doses of neutralising monoclonal antibody CAP256V2LS administered with and without VRC07-523LS in HIV-negative women in South Africa (CAPRISA 012B): a phase 1, dose-escalation, randomised controlled trial. ” Clinical trials are underway. Ricciardi M.J. The Lancet HIV.

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Agency IQ

DECEMBER 1, 2023

FDA’s guidance on developing products to prevent or treat Covid-19 Of the five guidance documents that received an extension, one addresses the development of drugs and biological products for Covid-19. Lastly, the revised guidance briefly discussed trials meant to assess drugs intended for prevention of Covid-19.

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The Pharma Data

FEBRUARY 25, 2021

The FDA approved Evrysdi in August 2020 as the first and only at home SMA treatment with proven efficacy in adults, children and infants 2 months and older. Evrysdi has been and continues to be studied in more than 450 people as part of a large and robust clinical trial program in SMA. mg/kg for Part 2.

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The Pharma Data

SEPTEMBER 27, 2020

In August, the FDA approved Evrysdi for the treatment of SMA in adults and children 2 months and older. The FDA approved Evrysdi for the treatment of SMA in adults and children 2 months of age and older. 59% of infants were able to sit without support for at least 5 seconds. No new safety signals were identified.

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The Pharma Data

FEBRUARY 8, 2021

Patients in the trial received either a combination of ERLEADA ® and ZYTIGA ® plus prednisone (combination arm) or placebo and ZYTIGA ® plus prednisone (control arm). [i] Food and Drug Administration (FDA) approval for nmCRPC on February 14, 2018 and was approved for mCSPC on September 17, 2019. All rights reserved.

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The Pharma Data

JANUARY 26, 2021

“Notably, the 70 percent decrease in risk of hospitalizations or death seen in this Phase 3 trial of bamlanivimab and etesevimab together is consistent with the reduction in risk of hospitalization or ER visits seen with bamlanivimab alone in the Phase 2 trial. INDIANAPOLIS, Jan. Across 1,035 patients, there were 11 events (2.1

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The Pharma Data

MAY 4, 2021

See Warnings and Precautions in the FDA-approved full Prescribing Information for additional information on risks associated with longer-term treatment with baricitinib. It is approved in the U.S. Olumiant was recently approved in Japan for the treatment of pneumonia associated with COVID-19 in hospitalized adult patients.

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Metabolite Tales Blog

JANUARY 22, 2025

Metabolism of 2024 FDA approved small molecules part 1 By Julia Shanu-Wilson Involvement of active metabolites The number of small molecules approved by the FDA in 2024 totaled 31 out of 50 NMEs with 56% (28) of these comprising small molecule therapies. Clin Pharmacokinet. What’s next? 2015 May;54(5):457-71.

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Common Sense for Drug Policy Blog

OCTOBER 29, 2024

Limited Evidence for Nalfmefene "In 2021, due to the widespread availability of high-potency synthetic opioids like fentanyl, the US FDA approved two high-dose naloxone products, an 8 mg IN spray (Kloxxado) and a 5 mg IM injectable (Zimhi). In 2023, the FDA approved a 2.7 In 2023, the FDA approved a 2.7

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The Pharma Data

APRIL 26, 2022

Learn more about FDA-approved and authorized COVID-19 vaccines. Given the similar course of COVID-19 disease in adults and pediatric patients, today’s approval of Veklury in certain pediatric patients is supported by efficacy results from phase 3 clinical trials in adults.

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New Drug Approvals

APRIL 5, 2025

7] Treosulfan was authorized for medical use in the European Union in June 2019, [9] and approved for medical use in the United States in January 2025. [7] 14/L Trial II (NCT00822393), a randomized active-controlled trial comparing treosulfan to busulfan with fludarabine as a preparative regimen for allogeneic transplantation.

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Advarra

DECEMBER 14, 2022

Effective as of 2007, Medicare has offered coverage for routine costs in clinical trials. However, in order to be eligible for extended coverage under a clinical trial, several requirements must be met. Clinical trial agreement. However, this may continue to be of use to identify all costs associated with the clinical trial.

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