The Premier Consulting Blog

OCTOBER 11, 2022

However, acceptance of that data depends on a sponsor’s understanding of the FDA criteria for historical control groups, study data standards, and clinical meaningfulness. In this article, we review two case studies involving the successful use of RWD or RWE in advancing the clinical development of treatments for rare diseases.

Disease 52

Disease FDA FDA Approval Treatment 52

The Pharma Data

NOVEMBER 3, 2020

DEXTENZA is FDA approved for the treatment of ocular inflammation and pain following ophthalmic surgery. The most common non-ocular adverse event was headache (1%). Ocular Therapeutix’s first commercial drug product, DEXTENZA, is FDA-approved for the treatment of ocular inflammation and pain following ophthalmic surgery.

Clinical Trials 52

Clinical Trials FDA Approval Treatment Production 52

Advarra

JUNE 20, 2023

of new cancer drugs tested in Phase I were likely to receive Food and Drug Administration (FDA) approval. Additionally, in 2021, a literature review estimated the average capitalized research and development (R&D) costs per new cancer medicine at between $944 million and $4.54 This means study startup times can vary greatly.

Trials 52

Trials Clinical Trials FDA Approval Therapies 52

The Pharma Data

JANUARY 17, 2021

The primary endpoint of this trial is to evaluate patients for complete histological clearance of the tumor cells within the treated BCC lesion with secondary endpoints, evaluating subjects for investigational product treatment related adverse events, as well as serious adverse events, and cutaneous skin reactions.

Treatment 52

Treatment Trials FDA Approval RNA 52

The Pharma Data

JANUARY 7, 2021

Cerecor intends to use the net proceeds of the offering for general corporate purposes and working capital, primarily to support the ongoing clinical development of key assets within its pipeline and for general and administrative expenses. The company is also developing two monoclonal antibodies, CERC-002, and CERC-007.

Clinical Development 52

Clinical Development Disease Treatment FDA Approval 52

The Pharma Data

DECEMBER 3, 2020

No serious adverse events related to the vaccine candidates studied were reported. The majority of adverse events were mild and transient. We are proud to be partnered with Clover in our endeavors to develop a safe and effective COVID-19 vaccine that will be readily accessible around the world to combat this ongoing pandemic.”.

Vaccine 52

Vaccine Immune Response Virus RNA 52

The Pharma Data

JANUARY 13, 2021

Harpoon has four drug product candidates in clinical development for the treatment of solid and hematologic malignancies based on its proprietary TriTAC platform. In April 2020, Harpoon announced dosing of the first patient with HPN217 (BCMA TriTAC) in a Phase 1/2 clinical trial focused on relapsed/refractory multiple myeloma (RRMM).

Treatment 52

Treatment FDA Clinical Trials Trials 52

The Pharma Data

NOVEMBER 9, 2020

(Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for NVX-CoV2373, the Company’s COVID-19 vaccine candidate. NVX-CoV2373 (SARS-CoV-2 vaccine) FDA Approval History.

Vaccine 52

Vaccine FDA Clinical Trials Immune Response 52

The Pharma Data

AUGUST 19, 2021

The majority of treatment-related adverse events (AEs) were Grade 1-2. Vice President, Clinical Development, Solid Tumors, Janssen Research & Development, LLC. Vice President, Clinical Development, Solid Tumors, Janssen Research & Development, LLC. “We All events were Grade 1-2.

Treatment 52

Treatment FDA Approval Pharmacokinetics FDA 52

The Pharma Data

DECEMBER 17, 2020

Senior Vice President and Head of Global Clinical Development at Regeneron and lead author of the publication. patients under an Emergency Use Authorization, and we also continue a robust clinical development program.” In the initial 275 patients, rates of adverse events (AEs) were similar among groups.

Hospitals 52

Hospitals Production Clinical Trials Virus 52

NIH Director's Blog: Drug Development

JUNE 14, 2016

Waterworth notes that the findings come on the heels of early results released in March from a clinical trial testing the cardiovascular safety of the GLP1R-targeted drug Victoza® (liraglutide), which confirm the newly reported genetic findings [4]. 5] Clinical development success rates for investigational drugs. Novo Nordisk.

Drugs 52

Drugs Disease Drug Development Clinical Trials 52

The Pharma Data

JULY 6, 2021

billion in clinical development, regulatory and commercial launch-related milestone payments. Alector will lead the global clinical development of AL001 and AL101 through Phase 2 proof-of-concept. There are currently no FDA-approved treatment options for FTD. There are multiple heritable forms of FTD.

Disease 52

Disease Science Treatment Clinical Development 52

The Pharma Data

JANUARY 13, 2021

Fast Track designation facilitates the development and expedites the review of drugs that are intended to treat serious and life-threatening conditions and show the potential to fill an unmet medical need. Brilacidin for UP/UPS was licensed to Alfasigma S.p.A. in July 2019, who have recently initiated a Phase 1 study with their formulation.

Clinical Trials 52

Clinical Trials Trials Treatment Pharmaceuticals 52

The Pharma Data

OCTOBER 25, 2020

“We are pleased to continue pursuing additional neuroscience opportunities with BXCL501, targeting agitation associated with delirium, a fifth potential indication for this candidate and a condition for which there is no FDA-approved treatment,” commented Vimal Mehta, Chief Executive Officer of BTI.

Treatment 40

Treatment Trials FDA Hospitals 40

The Pharma Data

JUNE 26, 2021

Safety was assessed in 816 patients across all four Libtayo monotherapy pivotal trials in its approved indications. Adverse events were serious in 30% of patients and led to permanent discontinuation in 8% of patients. The extensive clinical program for Libtayo is focused on difficult-to-treat cancers.

Trials 52

Trials Treatment Therapies Disease 52

The Pharma Data

JANUARY 31, 2021

No serious adverse events related to the vaccine candidates studied were reported. These findings give confidence that Clover’s COVID-19 vaccine candidates are suitable for further clinical development.” Dynavax is a commercial stage biopharmaceutical company developing and commercializing novel vaccines.

Clinical Trials 52

Clinical Trials Vaccine Trials Virus 52

The Pharma Data

NOVEMBER 30, 2020

It showcases our ability to advance strategically important candidates against specific disease targets through early development to become attractive partnering opportunities in parallel with the multi-target deals we have established. ” Abbreviations used: CGRP – calcitonin gene-related peptide. About Sosei Heptares.

Small Molecule 40

Small Molecule Licensing Licensing Treatment 40

The Pharma Data

JUNE 28, 2021

Across all treatment arms, the most commonly reported adverse events were gastrointestinal-related. Injectable semaglutide 1 mg is a GLP-1 receptor agonist and the highest dose of injectable semaglutide FDA-approved for the treatment of type 2 diabetes. Treatment discontinuation rates due to adverse events were 5.1

Clinical Trials 52

Clinical Trials Treatment Trials FDA Approval 52

The Pharma Data

APRIL 11, 2021

In this cohort, the most common treatment-emergent adverse events of any grade (?20%) No patients in this cohort discontinued treatment due to treatment-related adverse events. ” In May 2020, Lilly’s first-in-class selective RET inhibitor Retevmo received Accelerated Approval from the U.S. Retevmo is an U.S.

Research 52

Research Treatment Therapies Trials 52

The Pharma Data

APRIL 8, 2021

Roche will present data from five studies from the EVRYSDI clinical development programme, which was designed to represent a broad spectrum of people living with SMA. The full range of data from Roche’s clinical development programme in neuroscience being presented at 2021 AAN include: Medicine and/or Therapeutic Area.

Clinical Trials 52

Clinical Trials Trials Disease Therapies 52

The Pharma Data

OCTOBER 30, 2020

Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to eight FDA-approved treatments and numerous product candidates in development, all of which were homegrown in our laboratories.

Hospitals 52

Hospitals Virus Production Clinical Trials 52

The Pharma Data

JANUARY 18, 2021

His research on understanding the role of angiogenesis and vascular endothelial growth factor (VEGF) in cancer development, led to the discovery that VEGF is a key mediator of angiogenesis associated with intraocular neovascular syndromes.

Science 40

Science Virus Treatment Disease 40

The Pharma Data

DECEMBER 15, 2020

is a biopharmaceutical company focused on the formulation, development, and commercialization of innovative therapies for diseases and conditions of the eye using its proprietary bioresorbable hydrogel-based formulation technology. The Company anticipates that subsequent events and developments will cause the Company’s views to change.

FDA Approval 40

FDA Approval Disease Therapies Clinical Development 40

The Pharma Data

JUNE 7, 2022

. “We look forward to providing an important new medicine and helping patients find the relief they so desperately seek from the varied and debilitating symptoms of this disease, contingent upon FDA approval.” Almirall also plans to submit these results this year to the European Medicines Agency (EMA) for authorization.

Trials 40

Trials Clinical Trials Licensing Licensing 40

The Pharma Data

DECEMBER 29, 2020

PRX-102 was well-tolerated in the study, with all adverse events being transient in nature without sequelae. The most common moderate treatment emergent adverse events were nasopharyngitis, headache and dyspnea. mL/min/ 1.73m 2 in males, and 86.14 mL/min/ 1.73m 2 in females and plasma lyso-Gb 3 mean levels were 51.81 nM and 13.81

Clinical Trials 52

Clinical Trials Disease Treatment Trials 52

The Pharma Data

DECEMBER 5, 2020

The most common adverse events (AEs) were cytokine release syndrome (CRS; 39%; n=19), anemia (37%; n=18), fatigue (35%; n=17), nausea (31%; n=15), pyrexia (31%; n=15) and back pain (27%; n=13). REGN5458 and odronextamab are currently under clinical development, and their safety and efficacy have not been evaluated by any regulatory authority.

Production 40

Production Trials Treatment Disease 40

The Pharma Data

DECEMBER 11, 2020

Appropriate medical treatment used to manage immediate allergic reactions must be immediately available in the event an acute anaphylactic reaction occurs following administration of Pfizer BioNTech COVID-19 Vaccine. BNT162b2 (SARS-CoV-2 vaccine) FDA Approval History. Source: Pfizer Inc. . Posted: December 2020.

Vaccine 52

Vaccine Clinical Trials Trials Licensing 52

The Pharma Data

DECEMBER 27, 2020

(NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today provided a year-end review and an outline of its plans for 2021. An additional objective this year will be to build greater awareness for our past achievements and our future plans. Source link.

Therapies 40

Therapies Clinical Trials Production Treatment 40

The Pharma Data

OCTOBER 28, 2020

Serious adverse events were numerically more frequent with placebo than REGN-COV2 treatment (0.8% Senior Vice President and Head of Global Clinical Development at Regeneron. Senior Vice President and Head of Global Clinical Development at Regeneron. REGN-COV2 was well tolerated in the trial. high dose, 1.6%

Virus 40

Virus Trials Clinical Trials Production 40

The Pharma Data

NOVEMBER 20, 2020

Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments. BNT162b2 (SARS-CoV-2 vaccine) FDA Approval History. The information contained in this release is as of November 20, 2020. Source: Pfizer Inc. . Source link.

Vaccine 52

Vaccine FDA Clinical Trials Trials 52

The Pharma Data

NOVEMBER 9, 2020

Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments. BNT162b2 (SARS-CoV-2 vaccine) FDA Approval History. The information contained in this release is as of November 9, 2020. Source: Pfizer, inc. Posted: November 2020.

Vaccine 40

Vaccine Clinical Trials Trials FDA 40

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. This involves mindful messaging that the agency does not have traditional safety concerns regarding adverse events.

Science 40

Science FDA Clinical Trials Clinical Pharmacology 40

The Pharma Data

NOVEMBER 24, 2020

FDA Approves Oxlumo (lumasiran) for the Treatment of Primary Hyperoxaluria Type 1. Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients.

FDA Approval 52

FDA Approval Treatment FDA RNA 52

The Pharma Data

NOVEMBER 20, 2020

FDA Approves Zokinvy (lonafarnib) for Hutchinson-Gilford Progeria Syndrome and Processing-Deficient Progeroid Laminopathies. Nasdaq:EIGR), focused on the development and commercialization of targeted therapies for serious rare and ultra-rare diseases, today announced that the U.S. PALO ALTO, Calif., Outside the U.S.,

FDA Approval 52

FDA Approval FDA Clinical Trials Disease 52

The Pharma Data

OCTOBER 14, 2020

WAKIX is the first and only treatment approved by the FDA for people with excessive daytime sleepiness or cataplexy associated with narcolepsy that is not scheduled as a controlled substance by the U.S. WAKIX received FDA approval for the treatment of excessive daytime sleepiness in adult patients with narcolepsy in August 2019.

FDA Approval 40

FDA Approval FDA Treatment Clinical Trials 40

The Pharma Data

JULY 15, 2021

Merck Has Submitted These Data From KEYNOTE-522 to the US FDA for Review. Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced positive event-free survival (EFS) data from the pivotal neoadjuvant/adjuvant Phase 3 study KEYNOTE-522. 10) as determined by an FDA-approved test.

Therapies 52

Therapies Treatment Trials FDA 52

The Pharma Data

MARCH 28, 2022

Ozempic ® is now approved in the US at 0.5 Further, Ozempic ® is indicated to reduce the risk of major cardiovascular events such as heart attack, stroke or death in adults with type 2 diabetes and known heart disease. The FDA approval is based on the results from the SUSTAIN FORTE trial. mg and 2.0 Ozempic ® 2.0

Treatment 52

Treatment FDA Approval Trials FDA 52