2019 and FDA Approval - Drug Discovery Digest

Tofersen

New Drug Approvals

MAY 17, 2025

Jump up to: a b c d e f g h i j k l “FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene” (Press release). Food and Drug Administration (FDA). Jump up to: a b New Drug Therapy Approvals 2023 (PDF). Food and Drug Administration (FDA) (Report). January 2024.

FDA Approval

FDA Approval FDA Treatment Clinical Trials

Prime editing treats childhood brain disease in mice

Broad Institute

JULY 21, 2025

Prime editing was developed in 2019 by the lab of David Liu , a Broad core institute member and co-senior author of the new paper. The scientists delivered the prime editors to cells in mice using clinically validated viruses called AAVs, which are already used in FDA-approved gene therapies targeting brain cells.

Disease

Disease Laboratories Treatment DNA

Vamorolone

New Drug Approvals

JULY 25, 2025

Syn EuropeanJournalofMedicinalChemistry265(2024)116124 Vamorolone (Agamree) On October 26, 2023, Vamorolone, developed jointly by Santhera Pharmaceuticals and ReveraGen BioPharma, has received FDA approval to treat DMD in patients aged 2 years and older [1]. Alliance 2019, 2, No. Alliance 2019, 2,No. February 2019).

FDA

FDA FDA Approval Treatment Clinical Trials

Gepirone

New Drug Approvals

JUNE 11, 2025

However, in 2012 it once again failed to convince the FDA of its qualities for treating anxiety and depression. [5] 5] In December 2015, the FDA once again gave gepirone a negative review for depression due to concerns of efficacy. [12] 12] However, in March 2016, the FDA reversed its decision and gave gepirone ER a positive review. [13]

FDA

FDA FDA Approval Treatment Clinical Trials

Inavolisib

New Drug Approvals

APRIL 25, 2025

3] Inavolisib was approved for medical use in the United States in October 2024. [3] 19] Society and culture Legal status In October 2024, the US Food and Drug Administration (FDA) approved inavolisib for the treatment of PIK3CA -mutant breast cancer based on the results from the INAVO120 trial. [3] 20 May 2019.

FDA

FDA FDA Approval Therapies Trials

Elacestrant

New Drug Approvals

MAY 9, 2025

2] Elacestrant was approved for medical use in the United States in January 2023, [1] [2] [5] [6] and in the European Union in September 2023. [3] 2] The FDA granted the application for elacestrant priority review and fast track designations. [2] Food and Drug Administration (FDA). 1] [4] It is taken by mouth. [1]

FDA

FDA Pharmacokinetics Therapies Treatment

A Visual Guide to Gene Delivery

Codon

JULY 28, 2025

Casgevy , the first FDA-approved CRISPR-based therapy, is also an ex vivo gene therapy; it aims to cure sickle cell disease by editing a patient’s blood-producing stem cells so they generate functional fetal hemoglobin, then returning these corrected cells to the bloodstream.

Immune Response

Immune Response DNA Therapies Virus

A Visual Guide to Genome Editors

Codon

JUNE 5, 2025

.” Four years earlier, in 2019, Gray had become the first patient with sickle cell anemia — a genetic disorder that causes red blood cells to become sticky and rigid — to receive an experimental treatment using CRISPR genome editing. Dozens more clinical trials, based upon similar gene-editing technologies, are now underway.

DNA

DNA RNA Clinical Trials Engineering

Crinecerfont

New Drug Approvals

APRIL 18, 2025

2] Crinecerfont was approved for medical use in the United States in December 2024. [2] 2] [3] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [4] 2] The FDA granted the approval of Crenessity to Neurocrine Biosciences, Inc. [2] Food and Drug Administration (FDA) (Press release).

FDA

FDA FDA Approval Drugs Trials

Landiolol

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 FDA Approves AOP Health’s Rapiblyk (landiolol) for Atrial Fibrillation and Atrial Flutter in the Critical Care Setting” (Press release). 24 August 2022. AOP Health.

FDA

FDA Clinical Trials Treatment Trials

Medicare program spent $1.8 billion in 2019 on drugs without confirmed clinical benefits

The Pharma Data

MAY 27, 2022

billion in Medicare funds in 2019 on drugs whose clinical benefits have yet to be confirmed by the Food and Drug Administration, a new study led by researchers from the Johns Hopkins Bloomberg School of Public Health suggests. billion on 36 of these drugs across 55 indications in 2019. federal government spent an estimated $1.8

Drugs

Drugs Clinical Trials FDA Government

Roche announces FDA approval of FoundationOne Liquid CDx, a comprehensive pan-tumour liquid biopsy test

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. A negative result does not rule out the presence of an alteration.

FDA Approval

FDA Approval FDA DNA Therapies

Metabolism of 2022 FDA approved small molecule drugs PART 1

Metabolite Tales Blog

JANUARY 26, 2023

Metabolism of 2022 FDA approved small molecule drugs – Part 1 Does CYP3A4 still rule? By Julia Shanu-Wilson It won’t come as much surprise to learn that of the 17 small molecules* approved by the FDA in 2022, CYP3A4 was the major player in drug metabolism. 2019), although many of these are very minor.

Small Molecule

Small Molecule FDA Approval FDA Drugs

FDA Approves Pfizer Med for Rare Form of Pediatric Non-Hodgkin Lymphoma

The Pharma Data

JANUARY 14, 2021

Food and Drug Administration (FDA) approved Pfizer ’s Xalkori (crizotinib) for pediatric patients one year of age and older and young adults with relapsed or refractory, systemic anaplastic large cell lymphoma (ALCL) that is anaplastic lymphoma kinase (ALK)-positive. Manuel Esteban/Shutterstock.

FDA Approval

FDA Approval FDA Therapies Treatment

Metabolism of 2022 FDA approved small molecule drugs PART 2

Metabolite Tales Blog

APRIL 4, 2023

Metabolism of 2022 FDA approved small molecule drugs part 2 Mixing it Up By Julia Shanu-Wilson In Part 1 of this topic we looked at metabolism of the small molecule drugs approved by the FDA in 2022 that were mediated by CYP3A4. We hope it was a useful two-parter! We’re looking forward to the next crop! 6] Grillo et al.,

Small Molecule

Small Molecule FDA Approval FDA Drugs

FDA Approves Klisyri (tirbanibulin) for the Treatment of Actinic Keratosis on the Face or Scalp

The Pharma Data

DECEMBER 15, 2020

FDA Approves Klisyri (tirbanibulin) for the Treatment of Actinic Keratosis on the Face or Scalp. Food and Drug Administration (FDA) has approved Klisyri (tirbanibulin) for the topical treatment of actinic keratosis (AK) on the face or scalp. The FDA approval of Klisyri is a significant milestone for Athenex.

FDA Approval

FDA Approval Treatment FDA Therapies

FDA approves GlaxoSmithKline’s Nucala to treat hypereosinophilic syndrome

The Pharma Data

SEPTEMBER 28, 2020

Nucala was first approved in 2015 to treat severe eosinophilic asthma as an add-on maintenance therapy for the condition, and for the treatment of eosinophilic granulomatosis with polyangiitis. It also received approval in 2019 to treat severe eosinophilic asthma in children. .

FDA Approval

FDA Approval FDA Treatment Therapies

Kite Receives U.S. FDA Approval of New State-of-the-Art CAR T-Cell Therapy Manufacturing Facility in Maryland

The Pharma Data

APRIL 19, 2022

Food and Drug Administration (FDA) has approved commercial production at the company’s new CAR T-cell therapy manufacturing facility in Frederick, Maryland. The site will produce Kite’s FDA approved CAR T-cell therapy used to treat blood cancer.

FDA Approval

FDA Approval Therapies FDA Hospitals

Mylan secures first FDA approval for generic version of Biogen's Tecfidera following patent dispute

The Pharma Data

AUGUST 20, 2020

The approval follows just one month after Mylan triumphed in the US District Court for the Northern District of West Virginia to invalidated Biogen’s patent on Tecfidera, though the company is appealing the decision. billion for Biogen through 2019. The drug generated $3.79 Matt Fellows. Source link.

FDA Approval

FDA Approval FDA Treatment Production

TaiGen Announces FDA Approval of IND for Its Flu Antiviral TG-1000

The Pharma Data

OCTOBER 31, 2020

With the IND approval in place, we are ready and eager to extend the clinical study of TG-1000 to the U.S. billion USD in 2019 and is estimated to reach 5.03 in the near future.” ” According to Global Data, the global market for influenza antivirals reached 2.34 billion USD by 2026 at a CAGR of 11.5%.

FDA Approval

FDA Approval FDA Virus Clinical Development

Advanced Bionics Receives FDA Approval for Marvel Cochlear Implant Platform and World’s First Sound Processor for Children

The Pharma Data

DECEMBER 9, 2020

.–( BUSINESS WIRE )– Advanced Bionics (AB) , a global leader in cochlear implant technology, in collaboration with Phonak, a leading provider of life-changing hearing solutions, receives FDA approval and announces it is bringing Marvel hearing technology to Advanced Bionics cochlear implant wearers. Gifford, R.

FDA Approval

FDA Approval FDA Production Licensing

Roche receives FDA approval for first HIV-1/HIV-2 Qualitative Test on the cobas 6800/8800 Systems in the fight against HIV/AIDS

The Pharma Data

AUGUST 31, 2020

Food and Drug Administration (FDA) approval for the cobas® HIV-1/HIV-2 Qualitative Test for use on the fully automated cobas® 6800/8800 Systems in the U.S. The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2019 employed about 98,000 people worldwide. In 2019, Roche invested CHF 11.7

FDA Approval

FDA Approval FDA Virus Disease

Optimising CRIPSR gene editing of hard-to-transfect cells

Drug Target Review

APRIL 12, 2024

This holds particular significance, as it is a prerequisite for FDA approval in biotechnology that any cell clones must originate from a single-cell progenitor. He was awarded with the Ignite Award at SLAS 2019. Georges has been an active member of the SLAS’ entrepreneurship and innovation scenes.

Cell Biology

Cell Biology FDA Approval FDA Research

Olutasidenib

New Drug Approvals

DECEMBER 19, 2022

FDA 12/1/2022, To treat adults with relapsed or refractory acute myeloid leukemia with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation, Rezlidhia Olutasidenib , sold under the brand name Rezlidhia , is an anticancer medication used to treat relapsed or refractory acute myeloid leukemia. [1] World Health Organization (2019).

FDA Approval

FDA Approval FDA Treatment Pharmaceuticals

Optimising CRISPR gene editing of hard-to-transfect cells

Drug Target Review

APRIL 12, 2024

This holds particular significance, as it is a prerequisite for FDA approval in biotechnology that any cell clones must originate from a single-cell progenitor. He was awarded with the Ignite Award at SLAS 2019. Georges has been an active member of the SLAS’ entrepreneurship and innovation scenes.

Cell Biology

Cell Biology FDA Approval FDA Research

Pfizer and BioNTech Initiate Rolling Submission of Biologics License Application for U.S. FDA Approval of Their COVID 19 Vaccine

The Pharma Data

MAY 7, 2021

The Pfizer-BioNTech COVID-19 Vaccine has not been approved or licensed by the U.S. Food and Drug Administration (FDA), but has been authorized for emergency use by FDA under an Emergency Use Authorization (EUA) to prevent Coronavirus Disease 2019 (COVID-19) for use in individuals 16 years of age and older.

Licensing

Licensing Licensing Vaccine FDA Approval

Researchers reprogram gene therapy viral vectors to bind specific protein targets

Broad Institute

JULY 19, 2023

Related news How one lab uses machine learning to solve a key gene therapy problem In 2019, Deverman’s group identified the protein that serves as a receptor for AAV-PHP.B, a capsid that is efficiently transported across the blood-brain barrier.

Therapies

Therapies Research Engineering Virus

FDA More Selective About Convening Advisory Committee Panels During Pandemic | 2021-01-18

The Pharma Data

JANUARY 18, 2021

Given the pandemic, 2020 can’t be viewed as a typical year, but Prevision noted that the number of applications for new drugs or indications sent to the committees had already decreased steadily pre-pandemic, with only 23 in 2019 and 30 in 2018. 9, 2020 ).

FDA

FDA FDA Approval Licensing Licensing

Kala Gets the Greenlight from FDA for Dry Eye Disease Treatment

The Pharma Data

OCTOBER 26, 2020

EYSUVIS is the first FDA-approved corticosteroid specifically for dry eye disease treatment. The FDA approved EYSUVIS based on the positive results from one Phase II and three Phase III trials. in January 2019 and is similar to EYSUVIS except at a higher 1% concentration of LE for post-surgery, versus.25%

Treatment

Treatment Disease FDA FDA Approval

Vanda-lay Litigation Industries, Inc.: Taking Stock of Vanda Pharmaceuticals, Inc.’s Big Bets on Petitioning and Litigation Against FDA and the Federal Government

FDA Law Blog: Biosimilars

OCTOBER 1, 2024

To that end, below is a list of the cases Vanda has filed over the past five years or so against FDA or another government agency or entity, as well as a list of FDA Citizen Petitions and appeals. FOOD AND DRUG ADMINISTRATION et al Challenge to FDA signatory authority on Tradipitant NDA action Memorandum Opinion (D.D.C.)

Government

Government FDA Pharmaceuticals FDA Approval

The Power of AI in Drug Discovery and Development

The Connected Lab

APRIL 8, 2020

For patients suffering from an illness with no approved treatment, the wait can be unnerving. To this day, more than 400 million people suffer from rare diseases and 95% of rare diseases lack an FDA approved treatment 3. Billion; Approval Rate for Drugs Entering Clinical Development Is Less Than 12%.” Nature , vol.

Pharma Companies

Pharma Companies Laboratories Drugs FDA Approval

Surely You Must be Kidding, PTO?!? “No, and Don’t Call Me Shirley!” – The Seemingly Slapstick (But Yet Unfunny) World of Recent Patent Term Extension Decisions (PART 1)

FDA Law Blog: Biosimilars

FEBRUARY 27, 2024

156, a patent may be extended only once (even if it would be eligible for extension on more than one occasion because it applies to several FDA-approved products), and only one patent may be extended for each regulatory review period. Another case of same-day (and same-time) FDA approvals! To that end, 35 U.S.C. § 156(c)(4)

FDA Approval

FDA Approval FDA Production Therapies

Otsuka beats back Novartis opposition with $15M purchase of smart pill developer Proteus

The Pharma Data

AUGUST 20, 2020

Through a collaboration first signed in 2012, the two companies in 2017 successfully won FDA clearance for the first digital pill, Abilify MyCite, an ingestible drug-device combination that can track whether a patient adheres to the Japanese drugmaker’s antipsychotic Abilify (aripiprazole). One potential reason for the struggle?

FDA Approval

FDA Approval FDA Production Marketing

Sumitovant to Snap Up Development Partner Urovant Sciences

The Pharma Data

NOVEMBER 15, 2020

Federal Drug Administration (FDA) for vibegron in December 2019 after reporting positive data from its 12-week Phase III EMPOWUR study. Vibegron also showed favorable longer-term efficacy, safety, and tolerability in a 40-week extension study. .

Science

Science Small Molecule FDA Approval Therapies

An Evolving Regulatory Environment for Rare and Orphan Diseases

Advarra

AUGUST 16, 2022

Food and Drug Administration (FDA) approval. Since then, the FDA has significantly changed its approach to rare and orphan diseases. The FDA Since 1983. The Orphan Drug Act of 1983 was instrumental in changing the number of orphan drugs approved in the U.S. Before 1983, only 38 orphan drugs had received U.S.

Disease

Disease Clinical Trials FDA Approval FDA

Motixafortide

New Drug Approvals

SEPTEMBER 20, 2023

Jump up to: a b “Aphexda approval letter” (PDF). World Health Organization (2019). Motixafortide 4F-benzoyl-TN-14003 BKT-140 BKT140 BL-8040 Protein Chemical FormulaC 97 H 144 FN 33 O 19 S 2 Protein Average Weight2159.6 8 September 2023. Archived from the original (PDF) on 14 September 2023. BioLineRx Ltd. 11 September 2023.

FDA Approval

FDA Approval FDA International Clinical Trials

Improving Drug Safety Through Cardiotoxicity Assessment

PerkinElmer

JANUARY 7, 2022

Sci Rep 9, 18911 (2019). link] New safety concerns identified for 1 in 3 FDA-approved drugs [Internet]. References Hingorani, A.D., Improving the odds of drug development success through human genomics: modelling study. 2021 [cited 16 December 2021].

Drugs

Drugs FDA Approval Pharmacokinetics Drug Development

Collegium Reports Net Income of $11.3 Million in the Third Quarter of 2020

The Pharma Data

NOVEMBER 5, 2020

resolving patent litigation brought in response to Teva’s Abbreviated New Drug Application, seeking approval to market a generic version of Xtampza ER prior to the expiration of Collegium’s applicable patents. FDA approval, and customary exceptions). million for the quarter ended September 30, 2019 (the “2019 Quarter”) and $33.6

Pharmaceuticals

Pharmaceuticals Production Marketing Pharmaceutical Companies

Data Presented at TCT Connect Finds Pre-PCI Use of Impella for AMI Cardiogenic Shock is Associated with Higher Survival, Particularly in Women

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

Hospitals FDA Approval FDA Treatment

Levacetylleucine

New Drug Approvals

APRIL 30, 2025

Jump up to: a b c d e f g h i j k l m n o “FDA Approves New Drug to Treat Niemann-Pick Disease, Type C” U.S. FDA Approval of Aqneursa for the Treatment of Niemann-Pick Disease Type C” IntraBio (Press release). Strupp M, Bayer O, Feil K, Straube A (February 2019). 24 September 2024. N Engl J Med.

FDA

FDA FDA Approval Disease Trials

Etrasimod

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] June 2019). 1] It is taken by mouth. [1] 20 (4): 413–420. 2017.03.007.

FDA

FDA FDA Approval International Pharmacokinetics

First And Only Randomized, Double-blind, Head-to-head Study Comparing Aimovig® (erenumab-aooe), An Anti-CGRP Pathway Therapy, To Topiramate Published In Cephalalgia

The Pharma Data

NOVEMBER 8, 2021

4,5 Aimovig is the first FDA-approved migraine preventive treatment that targets the CGRP receptor.6 About Aimovig® (erenumab-aooe) Aimovig is the first FDA-approved migraine preventive treatment that targets the calcitonin gene-related peptide (CGRP) receptor, which is associated with migraine.6

Therapies

Therapies FDA Approval Treatment Small Molecule

Artificial Intelligence (AI), Machine Learning (ML) and the FDA

The Premier Consulting Blog

NOVEMBER 12, 2023

In 2019, the FDA published a discussion paper on a proposed regulatory framework for modification to AI/ML-based software as a medical device (SaMD), which received a significant amount of feedback from stakeholders.

FDA

FDA Drug Development Regulations FDA Approval

Roche expands its multiple sclerosis portfolio with investigational BTK inhibitor fenebrutinib and initiates novel clinical trials for OCREVUS (ocrelizumab)

The Pharma Data

SEPTEMBER 8, 2020

Roche is therefore initiating two new Phase IIIb trials, one in RMS (named MUSETTE) and one in PPMS (named GAVOTTE), which will evaluate a higher OCREVUS dose compared with the currently approved 600 mg dose, with both evaluated at the twice-yearly (six-monthly) dosing schedule. In 2019, Roche invested CHF 11.7

Clinical Trials

Clinical Trials Trials Disease FDA Approval

Tofersen

Prime editing treats childhood brain disease in mice

Trending Sources

Vamorolone

Gepirone

Inavolisib

Elacestrant

A Visual Guide to Gene Delivery

A Visual Guide to Genome Editors

Crinecerfont

Landiolol

Medicare program spent $1.8 billion in 2019 on drugs without confirmed clinical benefits

Roche announces FDA approval of FoundationOne Liquid CDx, a comprehensive pan-tumour liquid biopsy test

Metabolism of 2022 FDA approved small molecule drugs PART 1

FDA Approves Pfizer Med for Rare Form of Pediatric Non-Hodgkin Lymphoma

Metabolism of 2022 FDA approved small molecule drugs PART 2

FDA Approves Klisyri (tirbanibulin) for the Treatment of Actinic Keratosis on the Face or Scalp

FDA approves GlaxoSmithKline’s Nucala to treat hypereosinophilic syndrome

Kite Receives U.S. FDA Approval of New State-of-the-Art CAR T-Cell Therapy Manufacturing Facility in Maryland

Mylan secures first FDA approval for generic version of Biogen's Tecfidera following patent dispute

TaiGen Announces FDA Approval of IND for Its Flu Antiviral TG-1000

Advanced Bionics Receives FDA Approval for Marvel Cochlear Implant Platform and World’s First Sound Processor for Children

Roche receives FDA approval for first HIV-1/HIV-2 Qualitative Test on the cobas 6800/8800 Systems in the fight against HIV/AIDS

Optimising CRIPSR gene editing of hard-to-transfect cells

Olutasidenib

Optimising CRISPR gene editing of hard-to-transfect cells

Pfizer and BioNTech Initiate Rolling Submission of Biologics License Application for U.S. FDA Approval of Their COVID 19 Vaccine

Researchers reprogram gene therapy viral vectors to bind specific protein targets

FDA More Selective About Convening Advisory Committee Panels During Pandemic | 2021-01-18

Kala Gets the Greenlight from FDA for Dry Eye Disease Treatment

Vanda-lay Litigation Industries, Inc.: Taking Stock of Vanda Pharmaceuticals, Inc.’s Big Bets on Petitioning and Litigation Against FDA and the Federal Government

The Power of AI in Drug Discovery and Development

Surely You Must be Kidding, PTO?!? “No, and Don’t Call Me Shirley!” – The Seemingly Slapstick (But Yet Unfunny) World of Recent Patent Term Extension Decisions (PART 1)

Otsuka beats back Novartis opposition with $15M purchase of smart pill developer Proteus

Sumitovant to Snap Up Development Partner Urovant Sciences

An Evolving Regulatory Environment for Rare and Orphan Diseases

Motixafortide

Improving Drug Safety Through Cardiotoxicity Assessment

Collegium Reports Net Income of $11.3 Million in the Third Quarter of 2020

Data Presented at TCT Connect Finds Pre-PCI Use of Impella for AMI Cardiogenic Shock is Associated with Higher Survival, Particularly in Women

Levacetylleucine

Etrasimod

First And Only Randomized, Double-blind, Head-to-head Study Comparing Aimovig® (erenumab-aooe), An Anti-CGRP Pathway Therapy, To Topiramate Published In Cephalalgia

Artificial Intelligence (AI), Machine Learning (ML) and the FDA

Roche expands its multiple sclerosis portfolio with investigational BTK inhibitor fenebrutinib and initiates novel clinical trials for OCREVUS (ocrelizumab)

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