Drug Discovery World

JANUARY 30, 2024

Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Lentivirus strategies are one of the most widely used tools in biomedical research and clinical testing, says Stella Vnook, CEO, Likarda. “Dr

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Drug Discovery World

MAY 6, 2024

The company is now investigating a promising gene therapy for Huntingdon’s disease in a Phase I/II trial, with patients already showing signs of preserved neurological function. The rights for etranacogene dezaparvovec were subsequently sold to CSL Pharmaceuticals. This pedigree has attracted beneficial international collaborations.

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Agency IQ

FEBRUARY 15, 2024

MHRA selects eight technologies to help it test its new innovative devices pathway The British regulator and its partners just released the list of products and companies chosen to participate in the pilot of its Innovative Device Access Pathway (IDAP). BY COREY JASEPH, MS, RAC | FEB 14, 2024 6:11 PM CST Quick background on the future U.K.

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Agency IQ

JUNE 28, 2024

FDA draft guidance on addressing misinformation clears White House review A few months ahead of a potential administration transition, the White House has cleared (with changes) a new question-and-answer draft guidance document focused on “addressing misinformation” about regulated life sciences products.

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Agency IQ

MAY 28, 2024

New FDA guidance on addressing misinformation under White House review A new question-and-answer draft guidance document focused on “addressing misinformation” about regulated life sciences products is currently under administrative review at the White House’s Office of Information and Regulatory Affairs (OIRA).

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SugarCone Biotech

JUNE 10, 2024

This is where things get interesting due to the complexity of the immune system and its regulation. 4-1BB enhances the response of activated T-cells to “foreign antigens”: protein fragments derived from infected, diseased or cancerous cells that are presented to T cells by antigen-presenting cells (APC). Let’s back up a bit.

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Agency IQ

JUNE 2, 2023

What We Expect the FDA to do in June 2023 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the month ahead, including key deadlines, meetings, events, planned regulations, comment periods and more. An updated Unified Agenda: The Unified Agenda is the U.S.

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Agency IQ

JULY 12, 2024

BY LAURA DIANGELO, MPH | JUL 10, 2024 3:47 PM CDT Regulatory background: The FDA is responsible for overseeing information about regulated products. In regulation, intended use is defined at 21 CFR 201.128 for drugs and 21 CFR 801.4 sponsors, developers and manufacturers] to disseminate information about drugs and devices.”

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FDA Law Blog: Biosimilars

JUNE 27, 2024

50,372 , 50,377 (June 13, 2024). DEA regulations also establish that “[t]he responsibility for the proper prescribing and dispensing of controlled substances is upon the prescribing practitioner, but a corresponding responsibility rests with the pharmacist who fills the prescription.” 21 C.F.R. §§ 1306.04(a), a), 1306.06.

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FDA Law Blog: Biosimilars

MARCH 4, 2024

By Véronique Li, Senior Medical Device Regulation Expert & Deborah L. Livornese — On February 6, 2024, FDA issued a draft guidance titled Notifying FDA of a Discontinuance or Interruption in Manufacturing of Finished Products or Active Pharmaceutical Ingredients Under Section 506C of the FD&C Act.

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FDA Law Blog: Biosimilars

FEBRUARY 29, 2024

156, as added by the 1984 Hatch-Waxman Amendments, for certain FDA-regulated products. 156 or case law that would support extension of the ‘929 patent that claims the product despite revocation of the biologics license application. Karst — Earlier this week, we posted Part 1 of our three-part series on U.S. That did not happen.

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Agency IQ

JANUARY 12, 2024

BY AMANDA CONTI | JAN 10, 2024 9:29 PM CST Quick background: Goal dates under the Prescription Drug User Fee Act (PDUFA) The FDA collects user fees as part of an essential bargain between regulators and industry. According to Downey, the timeline of pre-license inspections for biologics can create challenges.

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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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LifeSciVC

SEPTEMBER 15, 2022

For example, in DM1 we developed a novel disease model (hTfR1/DMSXL) that enabled us to evaluate DYNE-101 and demonstrate an impact on reducing nuclear foci and DMPK RNA, leading to splicing correction. Generating robust preclinical data to share with global regulators supports alignment on your trial design.

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Agency IQ

JUNE 21, 2024

CBER’s Peter Marks offers insights on a new Rare Disease Hub, upcoming accelerated approval guidance This week, PETER MARKS, director of FDA’s Center for Biologics Evaluation and Research (CBER), spoke at several sessions of the Drug Information Association (DIA) annual meeting.

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Agency IQ

JULY 22, 2024

Halfway there: Novel drug approvals and their supportive clinical trials so far in 2024 In the first half of 2024, the FDA’s Center for Drug Evaluation and Research (CDER) approved 21 novel drug products. Halfway through 2024: how are novel drug approvals shaping up?

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Agency IQ

DECEMBER 1, 2023

What We Expect the FDA to do in December 2023 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the month ahead, including key deadlines, meetings, events, planned regulations, comment periods and more.

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Agency IQ

FEBRUARY 15, 2024

166 guidance documents the FDA is actively working on in 2024 (and beyond) The FDA is set to be very, very busy in 2024. We’ve identified a total of 166 guidance documents that are under active development, and have the key details for you below.

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Agency IQ

MARCH 7, 2024

flu vaccines Yesterday, FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) weighed in on strains for seasonal influenza vaccines for the Northern hemisphere 2024-2025 season. Vaccines advisory panel endorses move to trivalent formulation for U.S. This means that each hemisphere has its own influenza season.

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Agency IQ

FEBRUARY 12, 2024

A closer look at CDER’s new 2024 guidance agenda The FDA’s Center for Drug Evaluation and Research, its drug review division, this week quietly updated its 2024 Guidance Agenda, a list of all draft guidance documents the agency is working on this calendar year. Read Agency IQ’s breakdown of the 2024 CDRH guidance agenda here].

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Codon

JULY 14, 2024

In 1940, Charles Drew—the first African-American researcher to earn a doctor of medical science degree at Columbia University—developed an ingenious method for separating and storing plasma , the liquid part of blood that contains essential proteins that promote clotting and regulate blood pressure. In the U.S.,

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Agency IQ

SEPTEMBER 29, 2023

What We Expect the FDA to do in October 2023 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the month ahead, including key deadlines, meetings, events, planned regulations, comment periods and more.

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Agency IQ

OCTOBER 27, 2023

What We Expect the FDA to do in November 2023 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the month ahead, including key deadlines, meetings, events, planned regulations, comment periods and more. This is what OIRA is currently reviewing.

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Codon

FEBRUARY 13, 2024

alone, according to the Centers for Disease Control and Prevention. Phage-based products have been licensed for use as antibacterial agents in the sanitation , food production , and animal health industries. It took two years to produce the phage cocktail to standards that regulators were happy with.

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Agency IQ

JUNE 29, 2023

What We Expect the FDA to do in July 2023 In this ongoing feature, AgencyIQ looks at public data to determine what the FDA is likely to do in the month ahead, including key deadlines, meetings, events, planned regulations, comment periods and more.

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Drug Target Review

FEBRUARY 28, 2024

Human γδ TCR repertoires in health and disease. Normality sensing licenses local T cells for innate-like tissue surveillance. Increased frequency of TCR gamma delta + T cells in disease-free survivors following T cell-depleted, partially mismatched, related donor bone marrow transplantation for leukemia. Cells 9(40):800 (2020).

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Codon

FEBRUARY 27, 2024

Most square centimeters of our built environments transmit next to no disease (think of walls, which we rarely so much as brush in passing), but some are touched with extraordinary frequency. Diarrheal diseases—attributed to viruses, such as noroviruses, which are spread via fomite transmission—killed about 1.5

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FDA Law Blog: Biosimilars

JANUARY 18, 2024

Rescheduling Recommendation, 2024 FDA’s 2023 analysis and recommendation finally came to light when Texas attorney Matt Zorn posted it on his website a week ago. FDA analyzed the following indications: anorexia related to a medical condition, anxiety, epilepsy, inflammatory bowel disease, nausea and vomiting (e.g.,

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Agency IQ

JUNE 16, 2023

Highlights of the EPA’s Unified Agenda for Spring 2023 The Office of Management and Budget has released the Unified Agenda for Spring 2023, which outlines the regulations that agencies expect to release in 2023 and 2024. This information would be used in considering the regulation of asbestos.

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Agency IQ

JULY 12, 2024

The 53 regulations that FDA is currently working on The FDA on Friday unveiled its much-anticipated Spring 2024 Unified Agenda, a document outlining the regulations the agency plans to release in 2024 and beyond. The latest update, announced this week, is known as the Spring 2024 Unified Agenda.

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Agency IQ

AUGUST 9, 2024

By Sebastian Godoy, MPH | Aug 6, 2024 5:32 PM CDT Background: Psychedelic drug development and regulatory context A growing body of evidence suggests that psychedelics may provide clinical benefit for certain purposes, especially mental health conditions. EMA has now published a report on the workshop; read on for AgencyIQ’s analysis.

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The Pharma Data

FEBRUARY 4, 2021

AbbVie announced positive top-line results from the Phase 3 ADVANCE and MOTIVATE studies, which evaluated the efficacy and safety of Skyrizi (risankizumab) for induction therapy in adult patients with moderate to severe Crohn’s disease (CD). The company expects continued strong total company sales growth leading up to the U.S.

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FDA Law Blog: Biosimilars

MAY 21, 2024

On Thursday, May 16, 2024 , the Attorney General issued a Notice of Proposed Rulemaking (“NPRM”) to initiate rulemaking proceedings to reschedule marijuana. The NPRM was signed by the Attorney General but published by DEA in the Federal Register on May 21, 2024. 44,597 (May 21, 2024). 802(16)) and regulated by 21 C.F.R.

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The Pharma Data

MAY 4, 2021

Rare Disease. TicoVac (Tick-borne Encephalitis Vaccine) — In February 2021, Pfizer announced that the FDA accepted for Priority Review a Biologics License Application (BLA) for its tick-borne encephalitis (TBE) vaccine, as submitted for active immunization to prevent TBE in individuals 1 year of age and older. Internal Medicine.

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Agency IQ

MAY 28, 2024

BY RACHEL COE, MSC | MAY 23, 2024 7:12 PM CDT Yesterday’s Energy & Commerce Health Subcommittee Hearing All three of FDA’s medical product center directors appeared before the Health Subcommittee of the U.S. Rare disease patients bounce all over the place before they get a diagnosis… Why do we continue to tolerate that? Thank you.

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Agency IQ

JULY 22, 2024

Facing headwinds, FDA moves ahead on diagnostics policy implementation but faces Congressional, legal, and judicial questions In April 2024, the FDA issued a final rule to update its regulatory approach to certain test products known as Laboratory Developed Tests (LDTs). AgencyIQ provides a status update for regulated industry.

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Agency IQ

JULY 28, 2023

October 1, 2023 [link] Regulations Expected to be Published in Q3These are taken from the most recent version of the White House’s Unified Agenda. Specifically, this ANPRM seeks comments, research, information, data, and responses from certain categories of stakeholders that can inform the Agency’s modernization of FDA’s recall regulation.

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