The Pharma Data

NOVEMBER 21, 2021

People with achondroplasia have a genetic mutation that causes a certain growth regulation gene called fibroblast growth factor receptor 3 to be overly active, which prevents normal bone growth. A condition of this accelerated approval is a post-marketing study that will assess final adult height. Related Information.

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FDA Law Blog: Drug Discovery

DECEMBER 7, 2022

Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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The Pharma Data

NOVEMBER 27, 2020

FDA Approves Imcivree (setmelanotide) for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency. With this approval, Imcivree becomes the first-ever FDA approved therapy for these rare genetic diseases of obesity. Important Safety Information. BOSTON, Nov.

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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. The GAO Report further explained that the agency did not have the resources to regulate the estimated 100,000 OTC drugs marketed through the monograph process.

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Drug Target Review

MARCH 12, 2024

Bile acids are crucial signalling molecules that aid the regulation of the immune system and have essential metabolic functions, like controlling lipid and glucose metabolism. microbeMAAST: a taxonomically informed mass spectrometry search tool for microbial metabolomics data. Biochemical Pharmacology. Nature Microbiology.

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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. For more information on Kite, please visit www.kitepharma.com.

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The Pharma Data

JANUARY 15, 2021

Food and Drug Administration (FDA) approval of Darzalex Faspro ® (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, in combination with bortezomib, cyclophosphamide and dexamethasone (D-VCd) for the treatment of adult patients with newly diagnosed light chain (AL) amyloidosis.[1]

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The Pharma Data

FEBRUARY 24, 2022

See the prescribing information for additional information on risks associated with Jardiance. The FDA granted the approval of Jardiance to Boehringer Ingelheim. Related Information. FDA takes steps to spark development of heart failure drugs Heart Health for Women. ###. Source link: [link].

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The Pharma Data

NOVEMBER 12, 2021

Treatment is First FDA-Approved Option Patients Can Take Regardless of Previous Therapies. Food and Drug Administration approved Besremi (ropeginterferon alfa-2b-njft) injection to treat adults with polycythemia vera, a blood disease that causes the overproduction of red blood cells. Related Information. Today, the U.S.

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The Pharma Data

MARCH 15, 2022

To further facilitate generic drug development, and to assist the generic pharmaceutical industry in this process, the FDA publishes product-specific guidances (PSGs) describing the agency’s current thinking and expectations on how to develop generic drug products that are therapeutically equivalent to their brand name counterparts.

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Agency IQ

JUNE 2, 2023

FDA unveils long-awaited Patient Medication Information proposed rule Since 2017, the FDA has been working on a proposal to create a new type of patient-focused labeling for certain outpatient drug products that would be specifically targeted for patient use. The FDA recently concluded its work on a proposed rule focused on PMI.

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FDA Law Blog: Biosimilars

AUGUST 7, 2024

On July 30, brand name drug sponsor Novartis asked the District Court for a Temporary Restraining Order ( here and here ) enjoining FDA’s approval of a generic version of its ENTRESTO (sacubitril and valsartan) with certain dosing and indication information carved-out or modified.

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FDA Law Blog: Biosimilars

MARCH 3, 2024

The Act is intended to address national security concerns by prohibiting certain conduct by regulated industry. Effect on FDA approvals— In cases where a biological product or drug needs to change aspects of its manufacturing processes to avoid using a covered equipment or service, will it need to file supplements with FDA for the CMC update?

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The Pharma Data

DECEMBER 13, 2020

Food and Drug Administration (FDA) approved a first-of-its-kind intentional genomic alteration (IGA) in a line of domestic pigs, referred to as GalSafe pigs, which may be used for food or human therapeutics. The FDA granted approval of the IGA in GalSafe pigs to Revivicor Inc. SILVER SPRING, Md. ,

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Agency IQ

JUNE 16, 2023

The 51 regulations that FDA is currently working on The FDA today unveiled its much-anticipated Spring 2023 Unified Agenda, a document outlining the regulations the agency plans to release in 2023 and beyond. The anticipated date of publication is June 2023, meaning we should see this regulation imminently.

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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.

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The Pharma Data

MAY 17, 2021

. In a Viewpoint, Bishal Gyawali, MD, PhD, and colleagues at the Program on Regulation, Therapeutics and Law (PORTAL) research group at Harvard Medical School and Brigham & Women’s Hospital suggested improving FDA’s authority to modify drug package insert for situations such as drug repurposing and de-escalation of therapy.

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FDA Law Blog: Biosimilars

SEPTEMBER 30, 2024

Gaulkin — In May 2023, we posted about a CMS proposed regulation that sought to make a wide variety of changes to the Medicaid Drug Rebate Program (MDRP), including a new “price verification survey,” and a controversial proposal to require “stacking” of discounts to different customers when determining best price.

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DrugBank

MAY 6, 2024

A Wave of Innovations The year 2024 has already proven to be significant for the field of biologics, witnessing a notable increase in FDA approvals. In the first quarter alone, the FDA approved 9 new biologics, a substantial rise from the 5 approvals during the same period in 2023.

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FDA Law Blog: Biosimilars

OCTOBER 1, 2024

Specifically, Vanda alleges that FDA communications to certain generic competitors regarding dissolution rates, impurities, and micronization revealed Vanda’s confidential manufacturing information and caused economic injury to the company. FOOD AND DRUG ADMINISTRATION FOIA – FDA Presentation Pending 1:2023cv02325 (D.D.C.)

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The Premier Consulting Blog

DECEMBER 28, 2023

Under section §812.10, a sponsor may request a waiver of any requirement of the IDE regulations through an application with supporting documentation. The FDA may grant a waiver if the requirement is not stipulated by the Federal Food, Drug, and Cosmetic (FD&C Act) or if it is not necessary for the protection of human subjects. [3]

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The Pharma Data

MARCH 29, 2022

The agency will continue to evaluate data and information as it becomes available when considering the potential use of a second booster dose in other age groups. The FDA-authorized Pfizer-BioNTech COVID-19 Vaccine and the FDA-approved Comirnaty can be used to provide the authorized booster dose(s). Related Information.

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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. In several letters to applicants, each styled as a REQUIREMENT FOR INFORMATION PURSUANT TO 37 C.F.R.

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PPD

AUGUST 5, 2024

Food and Drug Administration (FDA)-approved therapies for treating PAH were primarily vasodilators, designed to overcome the imbalance between vasoactive and vasodilator mediators and to restore endothelial cell function. Historically, the available drugs and U.S.

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Drug Target Review

SEPTEMBER 19, 2023

This major shift to the orthodox tradition of using animal experiments in drug testing dates back the Aristotle’s time and cemented 80 years ago with initial federal mandate of drug safety regulation of 1938. To this end, the FDA’s newly created iSTAND initiative drives the path toward regulatory approval for devices like organ-on-chips.

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The Pharma Data

NOVEMBER 20, 2020

The FDA intends to make background materials available to the public, including the meeting agenda and committee roster, no later than two business days prior to the meeting. 23, the FDA intends to issue a Federal Register notice with details of the meeting, which will include information about a public docket for comments.

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FDA Law Blog: Biosimilars

AUGUST 12, 2024

As with other FDA-regulated products, such as human drugs and medical devices, the “regulatory review period” is composed of a “testing phase” and a “review phase.” The “review phase” is the period between the initial submission and approval of the NADA. FDA’s PTE regulations at 21 C.F.R.

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Broad Institute

JULY 26, 2022

Learn more Drug Repurposing Hub A curated and annotated collection of FDA-approved drugs, clinical trial drugs, and pre-clinical tool compounds. Learn more Genotype-Tissue Expression (GTEx) A comprehensive public resource to study tissue-specific gene expression and regulation.

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Pharmaceutical Development Group

AUGUST 9, 2021

Offshoring, sophisticated information technology, climate change, and a fresh approach to sales and marketing are all opportunities that the pharmaceutical sector may use. This condition of things is due to several variables. The pharmaceutical sector has certain distinct characteristics that influence product development.

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The Pharma Data

APRIL 7, 2023

“Fundamentally, however, the touchstone of FDA drug approval is a favorable benefit-risk assessment; without that favorable assessment, the drug should not have the status of being FDA-approved.” For additional information, see Makena Information on FDA.gov. Source link: [link]

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Advarra

NOVEMBER 17, 2022

In 2016, the Food and Drug Administration (FDA) approved Spinraza (nusinersen). While the FDA’s approval of nusinersen may not seem extraordinary, it was. Nusinersen’s approval marked the first time nonclinical data supported conducting initial clinical trials involving children. Why This Guidance Now?

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The Premier Consulting Blog

NOVEMBER 12, 2023

This feedback prompted the FDA to release a second paper in 2021 titled “ Artificial Intelligence/Machine Learning (AI.ML)-Based Software as a Medical Device (SaMD) Action Plan ,” which outlined the agency’s five-pillar approach to regulating AI/ML.

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The Premier Consulting Blog

OCTOBER 11, 2022

The treatment had been granted breakthrough therapy designation, but FDA approval would ultimately rest on the sponsor’s ability to demonstrate clinically meaningful improvement. Key Takeaway RWD and RWE play an important role in rare diseases, where capturing relevant data to support drug development is challenging.

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Advarra

JANUARY 23, 2024

Researcher Considerations for IRB Review of Study Changes Modification or amendment information should include sufficient detail for IRB assessment. The more information researchers provide in the IRB submission, the easier it is for the IRB to consider implications for the research, researcher, and current and future research participants.

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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] “FDA Approves New Drug for Ulcerative Colitis” Medscape.

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The Pharma Data

NOVEMBER 9, 2021

The State’s claims violate abecedarian principles of due process by seeking to hold a company liable for conduct permitted under civil law and regulations. Food and Drug Administration (FDA)-approved and since launch, have reckoned for lower than one percent of total opioid conventions in Oklahoma as well as the United States.

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Agency IQ

JULY 26, 2024

BY AMANDA CONTI | JUL 24, 2024 9:57 PM CDT Background: Modernizing regulation of over-the-counter (OTC) products Nonprescription products, also called over-the-counter (OTC) products, must demonstrate the ability to be used safely and effectively without the supervision of a qualified healthcare professional.

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