The Pharma Data

MAY 31, 2022

Food and Drug Administration in 2017. Evrysdi is currently approved in 81 countries and the dossier is under review in a further 27 countries. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement.

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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.

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Treatment Therapies Disease FDA Approval 52

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. .

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Science Small Molecule FDA Approval Therapies 52

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).

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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 52

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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). Otsuka, with 19.8%

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The Pharma Data

JANUARY 5, 2021

based contract manufacturing business, Benuvia Manufacturing, which has significant chemistry and formulation capabilities, including manufacturing our FDA-approved cannabinoid drug, SYNDROS ® ,” said Todd C. Investor Relations Contact: Peter Schwartzman Email: investor-relations@radiuspharm.com Phone: 617-583-2017.

Disease 52

Disease FDA Approval Clinical Trials Trials 52

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?

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Therapies Treatment Engineering Molecular Biology 59

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.

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Drug Development Treatment FDA Approval Drugs 52

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.

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Drug Target Review

SEPTEMBER 19, 2023

In spite of current success and possibility to be a successful cell technology model, the integration of organ-on-chips into drug development process needs more optimisation to be validated for FDA approval. Human computational models could provide faster, cheaper and additionally effective alternatives to experimental animal testing.

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Animal Testing Clinical Trials Pharmaceutical Companies FDA 98

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.

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Clinical Trials Trials RNA Bioinformatics 52

Eye on FDA

JANUARY 16, 2023

By contrast in 2021 there were only 10 (see blue bars below in the chart tracking meetings from 2017 – 2022). Yet in the midst of that range, the number of new molecular entities approved by FDA actually increased.

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Drug Target Review

JULY 5, 2024

Recently, a network-based drug-screening platform has been developed to test FDA-approved drugs for Alzheimer’s disease (AD) using 1,300 iPSC-derived organoids from 11 sporadic AD patients. 545:54-59 (2017). Cerebral organoids as tools to identify the developmental roots of autism. Molecular Autism. link] Birey F, et al.

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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]

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FDA FDA Approval Clinical Trials Treatment 52

NIH Director's Blog: Drug Development

JULY 20, 2017

The first major advance in molecularly targeted drug treatment for the disease came in 2012, when the Food and Drug Administration (FDA) approved ivacaftor (Kalydeco). Cystic Fibrosis Foundation News Release, July 18, 2017. About 30,000 Americans have CF. 1989 Sep 8;245(4922):1059-1065.

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Clinical Trials Small Molecule Therapies Disease 52

The Pharma Data

JANUARY 31, 2021

FDA approval for prevention of infection caused by all known subtypes of hepatitis B virus in adults age 18 years and older. CEPI is an innovative partnership between public, private, philanthropic, and civil organisations, launched at Davos in 2017, to develop vaccines to stop future epidemics. About CEPI.

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Clinical Trials Vaccine Trials Virus 52

The Pharma Data

DECEMBER 8, 2020

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. Pear’s reSET, reSET-O and Somryst are the first PDTs to receive FDA approval for treating disease. Headquartered in Shanghai, the company will also have a hub in Philadelphia.

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RNA Therapies Disease Protein Expression 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

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 52

Therapies Virus DNA Compliance 52

Drug Target Review

AUGUST 3, 2023

We use FDA-approved and CE-marked DNA collection devices that have been proved and tested as the gold standard in saliva-based DNA collection devices and are widely used by pharmaceutical companies and research institutions worldwide. Could you provide insights into the process of developing the at-home DNA tests used by Sano?

DNA 52

DNA Clinical Trials Trials Disease 52

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.

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FDA Regulations Clinical Trials Drugs 52

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. 8,128,929 [] for [PTE] request under 35 U.S.C. §

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Vial

JUNE 12, 2024

Role of PROs in drug approvals A recent review sought to comprehensively characterize the inclusion of PROs and regulatory considerations in FDA-approved novel oncology drugs.

Clinical Trials 52

Clinical Trials Trials FDA Clinical Research 52

The Pharma Data

DECEMBER 3, 2020

FDA approval for prevention of infection caused by all known subtypes of hepatitis B virus in adults age 18 years and older. CEPI is an innovative partnership between public, private, philanthropic, and civil organizations, launched at Davos in 2017, to develop vaccines to stop future epidemics. About CEPI.

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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. In 2017, the first year of the PDUFA VI agreement under the FDA Reauthorization Act, the fees were published on September 14, 2017.

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Agency IQ

MARCH 25, 2024

FDA addressed the expanding practice of drug compounding in 1992 by issuing a compliance policy guide that clarified that pharmacies which compounded products at certain scales, for certain purposes, or without FDA approval were clearly operating “outside the bounds of traditional pharmacy practice.” suppositories).

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The Pharma Data

SEPTEMBER 2, 2020

New Phase III data from SAkuraStar and SAkuraSky studies demonstrate reduced severity of relapses with ENSPRYNG (satralizumab), recently FDA-approved as the first and only subcutaneous treatment for adults living with anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD).

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Clinical Trials Treatment Disease Therapies 52

Practical Cheminformatics

AUGUST 3, 2023

Many papers I’ve read recently use the MoleculeNet dataset, released by the Pande group at Stanford in 2017, as the “standard” benchmark. Clintox – This dataset consists of 1483 SMILES strings and two binary labels indicating whether a molecule is an FDA-approved drug and whether a toxicity outcome has been reported.

Drugs 91

Drugs Biochemical Assays Metabolic Stability Screening Hits 91

The Pharma Data

DECEMBER 15, 2020

In September, Corbus Pharmaceuticals Holdings announced topline results from its 52-week Phase III RESOLVE-1 study of lenabasum in patients with diffuse cutaneous systemic sclerosis (SSc), a rare life-threatening multi-system autoimmune disease for which there are no FDA-approved treatments.

Disease 52

Disease Clinical Trials Small Molecule Trials 52

The Pharma Data

APRIL 8, 2021

Food and Drug Administration (FDA) approved EVRYSDI for the treatment of SMA in adults and children 2 months of age and older. EVRYSDI was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by FDA and EMA in 2017 and 2019, respectively.

Clinical Trials 52

Clinical Trials Trials Disease Therapies 52

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. The meeting’s outcome did not receive a rave review from former FDA Commissioner Scott Gottlieb, who led the agency between 2017 and 2019.

Science 40

Science FDA Clinical Trials Clinical Pharmacology 40

The Pharma Data

SEPTEMBER 10, 2021

Overview of the Population of Patients with Advanced Parkinson’s Disease (aPD) Initiating Therapy with Levodopa-Carbidopa Intestinal Gel (LCIG): POMPE-PARK Study on French Health Insurance Data (2013-2017). BOTOX ® Abstracts. OnabotulinumtoxinA Treatment in Patients with Upper Limb and Lower Limb Spasticity from the ASPIRE Study.

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Doctors Clinical Trials Disease Treatment 52

Drug Target Review

JUNE 21, 2023

2017 Mar 29;9(383):eaag1166. Human genetics evidence supports two-thirds of the 2021 FDA-approved drugs. 2017 Aug 3;101(2):177-191. 2017 Jun 8;546(7657):307-311. Epub 2017 May 31. The druggable genome and support for target identification and validation in drug development. Sci Transl Med. medRxiv; 2023.

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Broad Institute

OCTOBER 11, 2023

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. They are used to treat cancers including melanoma and non-small cell lung cancer, but work only for a small fraction of patients.

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FDA Law Blog: Biosimilars

AUGUST 21, 2023

Livornese — I saw the sign…and the answer is no—FDA-approved labeling apparently is not enough under state failure-to-warn laws, according to certain courts. That requirement would only be fulfilled if FDA agreed to such a change. But we digress. And indeed, after Pliva v.

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FDA Approval FDA Production Drugs 40

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.

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