Conversations in Drug Development Trends

DECEMBER 20, 2023

His presentation covered various aspects of oncology clinical programs, focusing on study design trends, with reference to both the recently implemented FDA Project Optimus guidance and studies we have seen from sponsors.

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The Pharma Data

FEBRUARY 25, 2022

Food and Drug Administration (FDA) has accepted for review the Prior Approval Supplement (PAS) to the Biologics License Application (BLA) for ABRILADA™ (adalimumab-afzb) as an interchangeable biosimilar to Humira® (adalimumab). The Biosimilar User Fee Act (BsUFA) goal date for an FDA decision is in Q4 2022. “An

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The Pharma Data

JUNE 7, 2022

New Indication for Amgen’s Fifth FDA-approved Biosimilar. The randomized, double-blind, comparative clinical study compared the efficacy, safety, pharmacokinetics and immunogenicity of RIABNI versus rituximab reference product (RP) in patients with moderate to severe RA. Now Approved to Treat All Available Rituxan ® Indications.

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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. Dual payload ADCs As effective as therapies have been in treating solid and haematological cancers, tumour heterogeneity and resistance remain major clinical challenges.

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New Drug Approvals

DECEMBER 24, 2023

“Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen” Neurology and Therapy. S2CID 250989659. ^ “Eplontersen: FDA-Approved Drugs” U.S. Food and Drug Administration (FDA). Ionis Pharmaceuticals.

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The Pharma Data

APRIL 27, 2021

Mobocertinib is the first oral therapy specifically designed to selectively target EGFR Exon20 insertion mutations. The application was submitted under the FDA’s accelerated approval program. and around the globe.”. Takeda has established an Expanded Access Program (EAP) ( NCT04535557 ) for patients in the U.S. In 2019, the U.S.

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PPD

OCTOBER 12, 2023

Cell and gene therapies (CGTs) are one of the fastest growing areas in human therapeutics. Since chimeric antigen receptor T cell (CAR-T) therapy was first approved in 2017, there has been a marked increase of cell and gene therapy studies resulting in significant changes in the way diseases are treated as well as patient outcomes.

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The Pharma Data

MAY 4, 2021

INDIANAPOLIS , May 4, 2021 /PRNewswire/ — Eli Lilly and Company (NYSE: LLY) is donating COVID-19 therapies to Direct Relief, enabling the humanitarian organization to provide COVID-19 therapies at no cost to low- and lower-middle-income countries most heavily impacted by the pandemic.

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The Pharma Data

MARCH 9, 2022

Sanofi and Swedish Orphan Biovitrum AB (publ) (Sobi ® ) (STO:SOBI) today announced positive topline results from the pivotal XTEND-1 Phase 3 study evaluating the safety, efficacy and pharmacokinetics of efanesoctocog alfa (BIVV001) in previously treated patients ? The US FDA granted Fast Track Designation in February 2021.

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Agency IQ

OCTOBER 20, 2023

FDA’s newest draft guidance lays out considerations for developing treatments for diabetic foot infections In a new draft guidance document released within weeks of a related clinical practice guideline, the FDA provides considerations for developing therapies to treat diabetic foot infections, focusing on Phase 3 efficacy trials.

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Alta Sciences

APRIL 17, 2024

Psychedelics Psychedelic therapy (or psychedelic-assisted therapy) refers to the use of psychedelic drugs, such as psilocybin, MDMA, LSD, ketamine, and ayahuasca, to treat mental disorders, especially those that have no effective treatments available or are treatment resistant.

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The Pharma Data

MARCH 9, 2022

PAXLOVID™ (nirmatrelvir [PF-07321332] tablets and ritonavir tablets) is the first oral therapy specifically designed to combat COVID-19 to be evaluated in a pediatric clinical study PAXLOVID is currently authorized under U.S. The safety and effectiveness of PAXLOVID have not yet been directly established in pediatric patients.

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LifeSciVC

APRIL 24, 2024

In the absence of a clinical trial result or FDA label to point to, how does one create the case and target product profile (TPP) around a new target? with gene editing or gene therapy, enzyme replacement therapy), agonism (e.g., in liver, in CNS)? with antibodies), or correction (e.g., in the case of CFTR for Trikafta).

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The Pharma Data

JULY 23, 2021

Food and Drug Administration (FDA) approved DALVANCE® (dalbavancin) for the treatment of acute bacterial skin and skin structure infections (ABSSSI) in pediatric patients from birth. 20% reduction in lesion size compared to baseline and no receipt of rescue antibacterial therapy for children 3 months and older.

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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] Food and Drug Administration (FDA). 1] It is taken by mouth. [1] April 2023).

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Advarra

JUNE 20, 2023

of new cancer drugs tested in Phase I were likely to receive Food and Drug Administration (FDA) approval. cell and gene therapies), with other therapeutic areas then pushing it further. Platform-type studies aiming to open and close cohorts quickly based on surrogate endpoints can help explore these targeted therapies more efficiently.

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Agency IQ

SEPTEMBER 22, 2023

BY AMANDA CONTI, ALEXANDER GAFFNEY, MS, RAC SEP 18, 2023 9:24 PM CDT Background: FDA’s standards for evidence When seeking approval from the FDA, companies are required to demonstrate that their product is safe and effective when used as intended. In one landmark case, Warner-Lambert Co.

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The Pharma Data

NOVEMBER 11, 2020

Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for SLV213 for the treatment of COVID-19 and has dosed the first subjects in a Phase 1 clinical study. Preclinical data show SLV213 is a potent inhibitor of SARS-CoV-2 infection. SAN DIEGO–( BUSINESS WIRE )– Selva Therapeutics, Inc.

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The Pharma Data

NOVEMBER 6, 2020

We appreciated the opportunity to share our data with the Advisory Committee, and we will continue to work with the FDA as it completes its review of our application.”. FDA Advisory Committees provide non-binding recommendations for consideration by the FDA. CAMBRIDGE, Mass. and TOKYO, Nov. About Alzheimer’s Disease.

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The Pharma Data

APRIL 4, 2023

Biosimilars help patients to gain broader access to effective and high-quality treatments that improve their disease therapies,” said Rebecca Guntern, Head of Region Europe, Sandoz. The study met all its primary objectives, demonstrating comparable pharmacokinetics and showing similar safety and immunogenicity between the two concentrations.

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Alta Sciences

AUGUST 28, 2023

The Phase I trial was performed to evaluate safety, tolerability, and pharmacokinetics in a group of 80 healthy participants in a two-part, double-blind, placebo-controlled study. The results of the trial showed a well-characterized pharmacokinetic profile, demonstrating dose proportionality over the ranges studied.

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The Pharma Data

NOVEMBER 26, 2020

Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for review of the Company’s Biologics License Application (BLA) seeking accelerated approval of pegunigalsidase alfa (PRX–102) for the proposed treatment of adult patients with Fabry disease. About Pegunigalsidase Alfa.

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The Pharma Data

JANUARY 26, 2021

percent) in patients taking therapy and 36 events (7.0 Bamlanivimab and etesevimab together also demonstrated statistically significant improvements on all key secondary endpoints, providing strong evidence that the therapy reduced viral load and accelerated symptom resolution. It remains under review by the FDA.

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The Pharma Data

APRIL 7, 2022

“At Pfizer, we have a strong heritage in, and commitment to, fighting infectious diseases, most recently evidenced by our delivery of the first authorized vaccine and oral therapy to combat COVID-19,” said Albert Bourla, Chairman and Chief Executive Officer, Pfizer. Food and Drug Administration (FDA). Sapir, CEO, ReViral.

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The Pharma Data

OCTOBER 25, 2020

Food and Drug Administration (“FDA”) for the treatment of agitation associated with delirium. Treatment choices are limited, and commonly used off-label therapies are not always effective or may result in prolonged, deep sedation. NEW HAVEN, Conn., 26, 2020 (GLOBE NEWSWIRE) — BioXcel Therapeutics, Inc. (“BTI”

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The Pharma Data

AUGUST 19, 2021

3] While MET inhibitors have recently received accelerated approval in this setting in some regions, the vast majority of patients eventually acquire resistance to these therapies, thus underscoring the need for new treatment options. [4] METex14 mutations are found in approximately three percent of patients with NSCLC. [2] 4] , [5] , [6].

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The Pharma Data

DECEMBER 29, 2020

Of the 22 patients enrolled in the BRIDGE study, the majority of treatment emergent adverse events were mild or moderate in severity, with two patients (9.1%) withdrawing from the therapy due to hypersensitivity reaction that was resolved. The most common moderate treatment emergent adverse events were nasopharyngitis, headache and dyspnea.

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Agency IQ

DECEMBER 11, 2023

The FDA and oncology: 2023 year in review As we round the corner into the last few weeks of 2023, AgencyIQ has taken a look back at a very busy year for the FDA’s oncology staff, and for sponsors. Since its inception, OCE has initiated an extensive list of initiatives and has taken the lead on over forty guidances published by FDA.

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Agency IQ

JUNE 9, 2023

FDA finalizes new guidance on nonclinical assessment of potential immunotoxicity Late last week the FDA released a final guidance document containing recommendations on the nonclinical assessment of potential immunotoxicity for drugs and certain biologics.

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The Pharma Data

DECEMBER 27, 2020

phase I trial is a randomized, double-blind, placebo-controlled, single and multiple dose escalation study to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics (biomarkers – FGF19 and C4) of ASC42 in healthy subjects. Food and Drug Administration (FDA) for NASH. ASC42 is an in-house developed?novel

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The Pharma Data

DECEMBER 15, 2020

The clinical responses were sustained by maintenance therapy with belimumab, an antibody to B-cell activating factor. The secondary outcome involves pharmacokinetic endpoints. The FDA has set an action date of July 7, 2021. The cells secrete auto-antibodies, but do not respond to standard immunosuppression. Receptor Inhibitors.

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The Pharma Data

JANUARY 19, 2021

This small molecule therapy is presently in Phase 1 clinical trial for mild to moderate Alzheimer’s disease (AD), which is supported by a NIA R01 grant in healthy aged volunteers. Alzheimer’s is a disease of aging, the greatest risk-factor, but there is not a single therapy available to halt or reverse this chronic neurodegenerative disorder.

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The Pharma Data

JULY 29, 2021

Food and Drug Administration (FDA) had granted Breakthrough Therapy designation for donanemab based on the Phase 2 data. Additionally, pharmacokinetic/pharmacodynamic modeling showed that greater relative amyloid plaque clearance was correlated with greater clinical benefit. In June 2021, Lilly announced the U.S.

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The Pharma Data

APRIL 14, 2021

government for its neutralizing antibody therapies authorized for emergency use as a treatment for COVID-19. Bamlanivimab and etesevimab together and bamlanivimab alone have not been approved by the FDA for any use. who require oxygen therapy due to COVID-19, OR.

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The Pharma Data

JANUARY 3, 2021

Food and Drug Administration (FDA) has cleared the Company’s Investigational New Drug (IND) application and that the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) has authorized its Clinical Trial Application (CTA) for a Phase 1/2 clinical trial evaluating INZ-701 in adults with ENPP1 deficiency.

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The Pharma Data

DECEMBER 21, 2020

As UNITY reported in its press release, this “Phase I, first-in-human, open-label, single-ascending dose study being conducted by UNITY is designed to evaluate the safety, tolerability, and pharmacokinetics of UBX1325 in patients with advanced DME. SUZHOU, China and ROCKVILLE, Md. , 22, 2020 /PRNewswire/ — Ascentage Pharma (6855.HK),

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The Pharma Data

APRIL 16, 2021

Food and Drug Administration (FDA) revoke the Emergency Use Authorization (EUA) for bamlanivimab (LY-CoV555) 700 mg alone. for the treatment of COVID-19 – as planned with the FDA – follows the modification of contracts with the U.S. Bamlanivimab and etesevimab together have not been approved by the FDA for any use.

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