New Drug Approvals

JULY 8, 2025

Clinical Trials: Pritelivir is currently in phase II clinical trials, with ongoing research into its effectiveness and safety. US FDA granted fast track designation for pritelivir in 2017 and breakthrough therapy designation 2020. PAPER By: Carta, Fabrizio ; et al.

Virus

New Drug Approvals

APRIL 19, 2025

Landiolol 133242-30-5 ONO-1101 Ono 1101 WHO 7516 FDA APPROVED 11/22/2024, Rapiblyk , To treat supraventricular tachycardia C25H39N3O8 509.6 Randomized clinical trials have been published to compare landiolol with placebo< [21] [22] [23] diltiazem, [24] and amiodaron [25] in patients with or without heart failure. July 2017).

FDA

The Pharma Data

JANUARY 18, 2021

Biederman, Director of Cardiovascular Imaging and Cardiac MRI at Allegheny General Hospital/Allegheny Health System in Pittsburgh and an author on the groundbreaking 2017 MagnaSafe trial published in NEJM. Typically, MRI is preferred by imaging specialists because it’s the safest alternative for most patients.

FDA Approval

The Pharma Data

AUGUST 27, 2020

The test is FDA-approved to report short variants in 311 genes including rearrangements and copy number losses in BRCA1 and BRCA2 genes. The results are delivered in an integrated report that identifies alterations matched to FDA-approved therapies.

FDA Approval

Vial

DECEMBER 5, 2023

In 2017, the Food & Drug Administration (FDA) approved the first gene therapy for cancer and for inherited diseases, the first multiplex NGS panel for companion diagnostics (CDx), and the first drug targeting a genetic signature though not a disease. Fountzilas et al.

Clinical Trials

Vial

JUNE 12, 2024

PROs in clinical trials are important as they capture the patient’s perspective and ensure that the impact of an intervention is comprehensively evaluated. Food and Drug Administration (FDA) increasingly look to patients to understand how they describe their health status. What are PROs in clinical trials?

Clinical Trials

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.

Treatment

The Pharma Data

MAY 31, 2022

.” Evrysdi is approved in 81 countries and the dossier is under review in a further 27 countries. More than 5,000 patients have now been treated worldwide with Evrysdi in clinical trials, compassionate use or real-world settings. Food and Drug Administration in 2017.

FDA Approval

The Pharma Data

JANUARY 31, 2021

Adjuvanted S-Trimer COVID-19 vaccine candidates demonstrated favorable safety and tolerability profiles and strong neutralizing immune responses in a phase 1 trial. Clover plans to initiate a global phase 2/3 trial in the first half of 2021 with an interim analysis for vaccine efficacy potentially in the middle of 2021.

Clinical Trials

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.

Disease

Drug Target Review

SEPTEMBER 19, 2023

The US FDA Modernisation Act 2.0., puts an end to the previous mandate that all drugs need to be tested on animals prior to human clinical trials. Human In Silico Drug Trials Demonstrate Higher Accuracy than Animal Models in Predicting Clinical Pro-Arrhythmic Cardiotoxicity. Nature Cancer. 2022;3(4):418-36.

Animal Testing

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] Peyrin-Biroulet L, Christopher R, Behan D, Lassen C (May 2017).

FDA

The Pharma Data

NOVEMBER 15, 2020

Our business is growing, and we remain focused on the potential opportunity to launch vibegron in 2021, pending FDA approval,” said Urovant Sciences President and chief executive officer, James Robinson. Vibegron also showed favorable longer-term efficacy, safety, and tolerability in a 40-week extension study. .

Science

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.

Disease

Drug Target Review

AUGUST 3, 2023

Clinical trials are now underway for various genetic subtypes of both rare and common diseases, on the understanding that medications utilising genetic biomarkers have a significantly higher chance of success. There are clinical trials underway for genetic subtypes of rare and common diseases.

DNA

The Pharma Data

OCTOBER 15, 2020

John Hospital, researchers compared 649 patients from two cohorts: a recent cohort (2017–2019) from the RECOVER III post-market approval (PMA) study, after the widespread adoption of the best practice of placing Impella pre-PCI, and a cohort from before PMA (2008–2014) when the practice of placing Impella pre-PCI was not yet widely adopted.

Hospitals

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. Asia, and Europe.

Drug Development

New Drug Approvals

AUGUST 26, 2023

Zuranolone CAS 1632051-40-1 Zurzuvae FDA APPROVED 8/4/2023, To treat postpartum depression Press Release WeightAverage: 409.574 Monoisotopic: 409.272927379Chemical FormulaC 25 H 35 N 3 O 2 SAGE 217 SAGE-217 SAGE217 Zuranolone , sold under the brand name Zurzuvae , is a medication used for the treatment of postpartum depression. [1]

FDA

NIH Director's Blog: Drug Development

JULY 20, 2017

Helping to fuel this optimism are preliminary results released this week from early-stage clinical trials of three next-generation, triple combination therapies that modulate the function of CFTR. According to preliminary data from one Phase 1 and two Phase 2 clinical trials reported by Vertex Pharmaceuticals Inc.,

Clinical Trials

The Pharma Data

DECEMBER 3, 2020

The Phase 1 trial was a randomized, observer-blind, placebo-controlled study to assess the safety, reactogenicity and immunogenicity of the adjuvanted COVID-19 S-Trimer vaccine candidates formulated with different antigen levels. The Phase 1 clinical trial was funded by the Coalition for Epidemic Preparedness Innovations (CEPI).

Vaccine

The Pharma Data

DECEMBER 8, 2020

Silverback’s lead candidate is currently in a Phase I trial in adults with HER2-expressing solid tumors. CEO and founder Min Li knows a thing or two from his time running GSK’s neuro R&D group before the company shuttered the program in 2017. Nuance Pharma .

RNA

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).

Clinical Trials

Drug Target Review

DECEMBER 5, 2024

1 In 2017, the US Food and Drug Administration (FDA) approved the first AAV-based gene replacement therapy (Luxturna), for Leber congenital amaurosis type 2. 2 Since then, the FDA has approved four more AAV-based gene therapies—Zolgensma, Hemgenix, Elevidys and Rocktavian—for treating various diseases.

Therapies

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. Small Molecule Inhibitors. The maximum achievable ACR CRISS score is 1.0.

Disease

The Pharma Data

SEPTEMBER 2, 2020

Initiation of Phase IIIb OCREVUS higher dose clinical trial programme and Phase IV study evaluating OCREVUS in minority populations. Roche is also deeply committed to addressing barriers to clinical trial participation and advancing inclusive research.

Clinical Trials

FDA Law Blog: Biosimilars

JULY 24, 2023

Approximately six weeks after FDA approved Lumryz and issued its clinical superiority decision , Jazz filed a Complaint against FDA in the District Court of D.C. Thus, Jazz argues, there is no evidence that Lumryz is more effective than Xywav.

FDA

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. That did not happen. The PTE application for U.S.

FDA

The Pharma Data

JUNE 18, 2021

BOTOX ® Cosmetic is the first and only product of its kind FDA-approved to treat three areas, temporarily improving the appearance of moderate to severe forehead lines, glabellar lines and crow’s feet in adults. Department of Health and Human Services FDA Approval Letter for BOTOX® Cosmetic for Glabellar Lines, 2002.

Doctors

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.

Science

The Pharma Data

SEPTEMBER 10, 2021

Levodopa/Carbidopa Intestinal Gel (LCIG) Reduces Fluctuations and Shortens Time to On Without Troublesome Dyskinesia in Advanced Parkinson’s Disease: Post-Hoc Analyses of 54-week LCIG-Monotherapy Trial. Adult Patients treated for overactive bladder due to neurologic disease: In clinical trials, 30.6% BOTOX ® Abstracts.

Doctors

Broad Institute

OCTOBER 11, 2023

By then, a few cancer immunotherapies had entered clinical trials and only a handful had been approved by the FDA. As a first step, Manguso set his sights on treatments called PD-1 checkpoint inhibitors, which at the time had shown tremendous promise in clinical trials and would receive their first FDA approval in 2014.

Research

Drug Target Review

JUNE 21, 2023

Strikingly, a strong genetic link between target and disease biology is emerging as a predictor for success in clinical trials. 2017 Mar 29;9(383):eaag1166. Why Clinical Trials Stop: The Role of Genetics. Human genetics evidence supports two-thirds of the 2021 FDA-approved drugs. 2017 Aug 3;101(2):177-191.

Disease

The Pharma Data

JUNE 18, 2021

Food and Drug Administration (FDA) has approved PREVNAR 20 (Pneumococcal 20-valent Conjugate Vaccine) for the prevention of invasive disease and pneumonia caused by the 20 Streptococcus pneumoniae (pneumococcus) serotypes in the vaccine in adults ages 18 years and older. Following today’s FDA approval, the U.S.

Vaccine

The Pharma Data

AUGUST 25, 2020

In August 2017, Bristol Myers Squibb received full approval in the U.S. Food and Drug Administration (FDA)-approved test. IDHIFA is the first and only FDA-approved therapy for patients with R/R AML and positive for an IDH2 mutation, which represents up to 19 percent of AML patients. WARNINGS AND PRECAUTIONS.

Trials

The Pharma Data

DECEMBER 18, 2020

The OLYMPUS, ALPS and ANDES trials evaluated roxadustat compared to placebo in NDD patients. Global, regional, and national burden of chronic kidney disease, 1990–2017: A systematic analysis for the Global Burden of Disease Study 2017. Related Articles: Roxadustat FDA Approval History. .

Disease

The Pharma Data

SEPTEMBER 28, 2021

Also, the info from SRP-9001 have helped to optimise the planning of the upcoming phase III clinical trial trial for DMD,” said Levi Garraway, M.D., quite 4,000 patients are treated with Evrysdi in clinical trials, compassionate use, and real-world settings. Roche’s Chief medic and Head of worldwide development.

Clinical Trials

The Pharma Data

AUGUST 22, 2021

Food and Drug Administration (FDA) for the adjuvant treatment of patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection, regardless of prior neoadjuvant chemotherapy, nodal involvement or PD-L1 status. months [95% Confidence Interval (CI): 16.5 months [95% CI: 8.3 to 13.9]).

FDA Approval