Metabolite Tales Blog

JANUARY 23, 2024

Metabolism of 2023 FDA Approved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. link] [19] FDA prescribing information for zilucoplan. link] [11] Zheng et al.,

Small Molecule 52

Small Molecule FDA Approval FDA Pharmacokinetics 52

The Pharma Data

MAY 18, 2021

Food and Drug Administration (FDA) approval of the VENTANA MMR RxDx Panel for advanced or recurrent endometrial cancer patients. FDA approval of the VENTANA MMR RxDx Panel provides clinicians with access to a fully automated, easy-to-use MMR test to identify patients who are eligible for therapy with JEMPERLI.

FDA Approval 40

FDA Approval FDA DNA Therapies 40

The Pharma Data

JUNE 18, 2021

First approval of a conjugate vaccine that helps protect against 20 serotypes responsible for the majority of invasive pneumococcal disease and pneumonia, 1,2,3,4,5,6,7 including seven responsible for 40% of pneumococcal disease cases and deaths in the U.S. Following today’s FDA approval, the U.S. Jansen, Ph.D.,

Vaccine 52

Vaccine FDA Approval FDA Disease 52

The Pharma Data

AUGUST 31, 2020

Detects, confirms and differentiates HIV-1 and HIV-2 infections providing clinicians with critical diagnostic data for personalised management of patients with HIV – from appropriate counseling on disease differences to targeted therapy. Centers for Disease Control and Prevention (CDC) estimated in 2018 that 1.2

FDA Approval 52

FDA Approval FDA Virus Disease 52

The Pharma Data

JANUARY 6, 2021

UNION therapeutics A/S is a privately held, clinical stage, pharmaceutical company dedicated to the development of novel treatments for inflammatory and infectious diseases. 2018)2: Menter MA, Armstrong AW, Gordon KB, Wu JJ. 2018 Feb;37(2S):S48-S51. About UNION therapeutics A/S. Sources 1: Li et al. Semin Cutan Med Surg.

FDA Approval 52

FDA Approval FDA Clinical Development Clinical Research 52

The Pharma Data

MAY 31, 2022

Without treatment, infants do not achieve these milestones in the natural history of the disease. “The priority review and subsequent approval of Evrysdi for babies under two months of age speaks to the urgent ongoing need for additional treatment options for babies with SMA,” said Levi Garraway, M.D.,

FDA Approval 52

FDA Approval FDA Treatment Clinical Trials 52

Codon

JULY 14, 2024

In contrast, people with sickle cell disease commonly receive red blood cell transfusions to alleviate vaso-occlusive pain crises. 2 And while transfusions are among the safest procedures in medicine, there is still a very slight risk of transmission of infectious diseases and adverse immune reactions. In the U.S.,

Production 133

Production Clinical Trials Trials Therapies 133

New Drug Approvals

SEPTEMBER 10, 2024

2] Vorasidenib was approved for medical use in the United States in August 2024. [2] 2] [3] It is the first approval by the US Food and Drug Administration (FDA) of a systemic therapy for people with grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 or isocitrate dehydrogenase-2 mutation. [2]

FDA Approval 62

FDA Approval FDA Clinical Trials Trials 62

Agency IQ

MAY 17, 2024

The Creating Hope Reauthorization Act would reauthorize the FDA’s Rare Pediatric Disease Priority Review Voucher program. The markup hearing comes three months after the E&C Committee last held a hearing on 18 bills related to rare diseases, including all four of the above-mentioned bills. DORIS MATSUI (D-Calif.)

FDA 40

FDA Disease FDA Approval Production 40

New Drug Approvals

SEPTEMBER 6, 2024

2 , 3 Lazertinib was first approved in South Korea on January 18, 2021, for the treatment of EGFR T790M mutation-positive non-small cell lung cancer (NSCLC) with EGFR mutations. 1 It was approved by the FDA on August 19, 2024. 1 It was approved by the FDA on August 19, 2024. Food and Drug Administration (FDA).

FDA Approval 48

FDA Approval FDA International Treatment 48

Drug Target Review

AUGUST 29, 2024

MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs. What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies?

Therapies 59

Therapies Treatment Engineering Molecular Biology 59

Eye on FDA

DECEMBER 6, 2023

For those working closely with the development of new medicines for FDA approval, it can be informative respecting the future to look back at recent activity and take note of any potential changes from years past. There are two things of note from this year.

FDA 77

FDA FDA Approval Therapies Drugs 77

The Pharma Data

APRIL 4, 2023

Food and Administration (FDA) approval for nmCRPC in February 2018, and received U.S. FDA approval for mCSPC in September 2019.To About the Janssen Pharmaceutical Companies of Johnson & Johnson At Janssen, we’re creating a future where disease is a thing of the past. Source link: [link]

Specialty Pharmacies 40

Specialty Pharmacies FDA Approval Pharmaceutical Companies Pharmacy 40

The Pharma Data

JUNE 17, 2022

The application includes all indications covered by the reference medicine*, including rheumatoid arthritis, Crohn’s disease, ulcerative colitis, plaque psoriasis, and uveitis 1. Sandoz’ Hyrimoz 50 mg/mL was first approved by the European Commission in July 2018 and launched in several European countries shortly thereafter.

Biosimilars 52

Biosimilars Pharmacokinetics FDA Approval Packaging 52

The Pharma Data

DECEMBER 30, 2020

In December, Gilead shifted from bolstering its oncology business and strengthened its liver disease pipeline with the $1.4 AstraZeneca Dives into Rare Diseases: AstraZeneca has been in the spotlight for the COVID-19 vaccine candidate it developed with Oxford University. Andexxa, was approved by the U.S. But the U.K.-based

Science 52

Science Disease Pharmaceuticals Treatment 52

Drug Target Review

JULY 5, 2024

Since brain organoids can be derived from either diseased or healthy patients, they can recapitulate human (patho)physiology making them a better choice compared to mammalian cell systems that often fail to recapitulate the human phenotype. 15(9):700-706 (2018) [link] Deshmukh V, et al. 12(3) (2023). link] Madhavan M, et al.

Targeted Protein Degradation 64

Targeted Protein Degradation Drugs Disease FDA Approval 64

New Drug Approvals

SEPTEMBER 8, 2024

1] Palopegteriparatide was approved for medical use in the European Union in November 2023, [2] and in the United States in August 2024. [1] 5] The FDA granted the application for palopegteriparatide orphan drug and priority review designations. [5] 4] [6] The applicant for this medicinal product is Ascendis Pharma Bone Diseases A/S.

FDA 62

FDA International Trials FDA Approval 62

The Pharma Data

AUGUST 19, 2021

1 Disease response was evaluated using overall response rate (ORR), per Response Evaluation Criteria in Solid Tumors Version 1.1* (RECIST v1.1) RYBREVANT TM (amivantamab-vmjw) received accelerated approval by the U.S. In 2018, Janssen Biotech, Inc. Interstitial Lung Disease/Pneumonitis 7. as the primary endpoint.

Treatment 52

Treatment FDA Approval Pharmacokinetics FDA 52

The Pharma Data

SEPTEMBER 28, 2021

Our goal is to still lead the way in developing transformative medicines for neuromuscular diseases. We are grateful for the partnerships that are helping us to develop new therapies for people impacted by these devastating rare diseases.”. About SMA SMA may be a severe, progressive neuromuscular disease which will be fatal.

Clinical Trials 52

Clinical Trials Protein Expression Therapies FDA 52

The Pharma Data

JANUARY 31, 2021

Many major disease targets are trimerization-dependent such as the tumor necrosis factor (TNF) superfamily (involved in extrinsic apoptosis, immune co-stimulation and inflammation) as well as enveloped RNA virus antigens responsible for entry into host cells. . CHENGDU, China , Feb. About Trimer-Tag© Technology.

Clinical Trials 52

Clinical Trials Vaccine Trials Virus 52

The Pharma Data

JUNE 30, 2021

Metastatic colorectal cancer is an aggressive disease with a poor prognosis, leaving patients with a critical need for additional treatment options beyond standard chemotherapy,” said Ian M. The Marketing Authorization approves use of Opdivo plus Yervoy in all EU member states, as well as Norway, Iceland and Liechtenstein. Waxman, M.D.,

Treatment 52

Treatment Trials FDA Approval Disease 52

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 52

DNA Clinical Trials Trials Disease 52

Dark Matter Blog

AUGUST 27, 2020

In early August, risdiplam, an orally available small molecule that binds to a stem-loop structure in the SMN2 pre-mRNA, received FDA approval for the treatment of spinal muscular atrophy , a devastating genetic disease. In less severe disease, patients will reach adulthood and suffer from motor function deficiencies.

Small Molecule 52

Small Molecule RNA Clinical Trials Drugs 52

The Pharma Data

DECEMBER 3, 2020

Many major disease targets are trimerization-dependent such as the tumor necrosis factor (TNF) superfamily (involved in extrinsic apoptosis, immune co-stimulation and inflammation) as well as enveloped RNA virus antigens responsible for entry into host cells. About Trimer-Tag © Technology. View source version on businesswire.com: [link].

Vaccine 52

Vaccine Immune Response Virus RNA 52

Codon

NOVEMBER 3, 2024

Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. But even now, more than a century later, TB remains the deadliest infectious disease on Earth, killing about 1.2 million people every year.

Vaccine 85

Vaccine Trials Therapies Treatment 85

The Pharma Data

MARCH 30, 2021

– Migraine is a debilitating neurological disease affecting 39 million people in the U.S.[2] Migraine is a complex, chronic disease with attacks that are often incapacitating and can include headache pain as well as neurologic and autonomic symptoms. AbbVie anticipates a regulatory decision in late Q3 2021.

Treatment 52

Treatment FDA Doctors FDA Approval 52

The Pharma Data

JANUARY 24, 2021

Sairiyo is a biotechnology company focused on repurposing and developing improved formulations of naturally derived compounds for serious, rare, and life-threatening diseases with the aim to obtain European Medicines Evaluation Agency and U.S. Food and Drug Administration (“FDA”) approval.

FDA 52

FDA Drugs Clinical Development Disease 52

Practical Cheminformatics

FEBRUARY 1, 2023

The 2018 paper by Gómez-Bombarelli, which launched the field, has already been cited more than 2,100 times. As of 2021, there are over 70 FDA-approved drugs that are kinase inhibitors. These drugs are used to treat a variety of conditions, including cancer, autoimmune disorders, and heart disease.

FDA Approval 52

FDA Approval FDA Drugs Science 52

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 52

FDA FDA Approval Clinical Trials Treatment 52

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 52

Doctors FDA Approval Treatment Production 52

The Pharma Data

FEBRUARY 8, 2021

Based on these data, ADT alone should no longer be considered sufficient for patients with advanced, castration-sensitive disease.”. Food and Drug Administration (FDA) approval for nmCRPC on February 14, 2018 and was approved for mCSPC on September 17, 2019. ERLEADA ® received U.S.

Treatment 52

Treatment Pharmaceutical Companies Therapies Pharmaceuticals 52

The Pharma Data

OCTOBER 25, 2020

Real-world effectiveness data on Oxbryta ® (voxelotor) in the treatment of sickle cell disease featured as oral presentation. Two abstracts have been accepted for presentation, including real-world effectiveness data of Oxbryta ® (voxelotor) tablets in the treatment of sickle cell disease (SCD). FDA approval. 26-31, 2020.

Treatment 40

Treatment Disease FDA FDA Approval 40

The Pharma Data

JUNE 18, 2021

“The results add to the extensive clinical evidence supporting the use of single-agent ibrutinib for long-term disease control.” IMBRUVICA ® is now approved in 101 countries and has been used to treat more than 230,000 patients worldwide across its approved indications. Barr , M.D., 1 Additionally, at 6.5

FDA Approval 40

FDA Approval Treatment Therapies FDA 40

Agency IQ

OCTOBER 6, 2023

This is a significant jump from any previous year, with the former peak at 15 in FY 2018, followed by several years in the 10-14 range. Taking a closer look, nearly half of the products were reviewed by the Office of Oncologic Diseases. FDA approved 13 NMEs through the AA pathway in FY 2023, making up 25.5%

FDA 52

FDA Biosimilars Science Production 52

The Pharma Data

JUNE 18, 2021

4,5 Between 20 and 46 percent of adults with atopic dermatitis have moderate to severe disease. 6 The range of symptoms pose significant physical, psychological and economic burden on individuals impacted by the disease. The approved dose for RINVOQ is 15 mg. Nat Rev Dis Primers 4, 1(2018). About RINVOQ ® (upadacitinib).

Treatment 40

Treatment Disease FDA Therapies 40

The Pharma Data

AUGUST 31, 2021

As the treatment of genitourinary cancers becomes more complex, we continue to work with urologists and their teams to improve outcomes for patients across the continuum of disease,” said Craig Tendler, M.D., Monitor for signs and symptoms of ischemic heart disease and cerebrovascular disorders. 2] ERLEADA ® received U.S.

Treatment 52

Treatment Trials FDA Approval Disease 52

The Pharma Data

MAY 18, 2021

5 percent of the tumour area) as determined by an FDA-approved test or are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. Tecentriq’s indication was subsequently focused on PD-L1 high patients, who would benefit the most based on findings from the IMvigor130 study in 2018.

FDA 40

FDA Treatment Pharmaceuticals Science 40