Drug Discovery World

SEPTEMBER 23, 2022

An interesting week for clinical research and drug approvals, with rare diseases being a key focus for both the FDA and European Commission. The last week has seen data emerge on how Omicron infection could protect against influenza, how breast cancer cells evade therapies, and two approvals on rare diseases in the US and EU.

FDA Approval 130

FDA Approval FDA Drugs Therapies 130

The Pharma Data

SEPTEMBER 7, 2020

Genentech’s once-daily oral therapy Gavreto (pralsetinib) has secured FDA backing in the treatment of metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), it has emerged. The decision was made under accelerated approval, marking Genentech’s sixth approval from the US regulator within lung cancer.

FDA Approval 52

FDA Approval FDA Clinical Trials Therapies 52

Drug Discovery World

NOVEMBER 25, 2022

Gene therapies have made the headlines over the last few days, with treatments for rare AADC deficiency, lung cancer and haemophilia B given the go ahead from regulators. FDA approves first disease-modifying therapy for T1D . FDA approves first gene therapy for haemophilia B .

FDA Approval 130

FDA Approval FDA Drugs Therapies 130

The Pharma Data

NOVEMBER 29, 2021

Cytalux is used with a Near-Infrared fluorescence imaging system cleared by the FDA for specific use with pafolacianine. The FDA previously granted Cytalux orphan-drug , priority and fast track designations. The FDA granted the approval to On Target Laboratories, LLC. The FDA, an agency within the U.S.

FDA Approval 52

FDA Approval FDA Drugs Laboratories 52

The Pharma Data

NOVEMBER 27, 2020

FDA Approves Danyelza (naxitamab-gqgk) for the Treatment of Neuroblastoma. Food and Drug Administration (“FDA”) has approved Danyelza (naxitamab-gqgk) 40mg/10ml. This indication is approved under accelerated approval regulation based on overall response rate and duration of response. NEW YORK, Nov.

FDA Approval 52

FDA Approval FDA Treatment Clinical Trials 52

FDA Law Blog: Biosimilars

AUGUST 7, 2024

Additionally, Novartis alleged that FDA unlawfully permitted the carve-out of critical safety information of a modified dosing regimen. Each of these issues, says Novartis, “independently renders the agency’s decision unlawful, and invalidates the agency’s approval of the MSN product.” From the government’s brief, see here.

FDA Approval 59

FDA Approval FDA Hospitals Regulations 59

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. The FDA, an agency within the U.S.

FDA Approval 52

FDA Approval FDA Regulations Drugs 52

The Pharma Data

AUGUST 13, 2020

NS Pharma has claimed a tentative FDA approval for its Viltepso (viltolarsen) injection in the treatment of Duchenne muscular dystrophy (DMD) in patients who are suitable to receive exon 53 skipping therapy. Based on these data, the FDA judged that Viltepso is “reasonably likely” to provide clinical benefit in the approved indication.

FDA Approval 52

FDA Approval FDA Therapies Hospitals 52

Drug Discovery World

NOVEMBER 14, 2023

A very rare, inherited blood clotting disorder, cTTP is caused by a disease-causing mutation in the ADAMTS13 gene, which is responsible for making an enzyme, also named ADAMTS13, that regulates blood clotting. A deficiency in this enzyme causes blood clots to form in the small blood vessels throughout the body.

Therapies 130

Therapies FDA Approval Protein Production FDA 130

Pharmaceutical Development Group

JANUARY 7, 2022

Effective and consistent use and application of Data Standards can reduce costs of Pharmaceutical Drug and Biologic Products and Process Development, Drug Development Services, 505(b) NDA, IND Consulting , NDA Consulting, BLA Consulting , and effective FDA Pre-Submission collectively resulting in FDA Approval. Spanogle, Ph.D.

FDA 52

FDA FDA Approval Biosimilars Regulations 52

FDA Law Blog: Biosimilars

FEBRUARY 26, 2024

Karst — While the Biologics Price Competition and Innovation Act (“BPCIA”) is inherently distinct from the Hatch-Waxman Act, many of the fundamental concepts FDA adopted as it enacted the Hatch-Waxman Act made their way into FDA’s implementation of the BPCIA. FDA borrowed this definition from 21 C.F.R.

FDA 59

FDA Biosimilars Production Regulations 59

The Pharma Data

MARCH 15, 2022

“Today’s approval of the first generic for one of the most commonly prescribed complex drug-device combination products to treat asthma and COPD is another step forward in our commitment to bring generic copies of complex drugs to the market, which can improve quality of life and help reduce the cost of treatment,” said Sally Choe, Ph.D.,

FDA Approval 52

FDA Approval FDA Production Pharmaceuticals 52

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 52

FDA Approval FDA Treatment Small Molecule 52

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. Kite, a Gilead Company (Nasdaq: GILD), today announced the U.S.

FDA Approval 52

FDA Approval Therapies FDA Hospitals 52

The Pharma Data

AUGUST 16, 2020

First and only FDA-approved subcutaneous treatment option for anti-aquaporin-4 antibody positive NMOSD that can be self-administered by a person with NMOSD or a caregiver every four weeks. First and only approved therapy for NMOSD designed to target and inhibit interleukin-6 receptor activity, using novel recycling antibody technology.

FDA Approval 52

FDA Approval FDA Clinical Trials Treatment 52

The Pharma Data

FEBRUARY 24, 2022

Food and Drug Administration approved Jardiance (empagliflozin) to reduce the risk of cardiovascular death and hospitalization for heart failure in adults. Jardiance was originally approved by the FDA in 2014 as a supplement to diet and exercise to improve glucose control in adults with type 2 diabetes. Related Information.

FDA Approval 52

FDA Approval FDA Treatment Hospitals 52

Agency IQ

JULY 8, 2024

In a new final rule, FDA carves out a regulatory niche for medical gases Industry has been lobbying FDA and Congress to regulate medical gases different from other types of drug products since the 1970s. Broadly, the rule sought to require the use of certain safeguards in the production, storage, and use of medical gases.

FDA 40

FDA Regulations Production Packaging 40

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. BOSTON, Nov. in the first quarter of 2021.

FDA Approval 52

FDA Approval FDA Clinical Trials Therapies 52

FDA Law Blog: Biosimilars

JANUARY 18, 2024

Palmer — A new lawsuit against FDA is the latest happening in the veterinary drugs space and, by extension, FDA’s Center for Veterinary Medicine (CVM). District Court challenging FDA’s plan to remove their products from the market. Specifically, FDA is looking to remove a drug called carbadox from the market. By John W.M.

FDA 59

FDA FDA Approval International Production 59

The Pharma Data

JULY 29, 2021

. “This is a momentous day for people who rely daily on insulin for treatment of diabetes, as biosimilar and interchangeable biosimilar products have the potential to greatly reduce health care costs,” said Acting FDA Commissioner Janet Woodcock, M.D.

Biosimilars 52

Biosimilars Production FDA Approval FDA 52

Drug Discovery World

MAY 21, 2024

Modern experimental procedures, such as immunisation, B-cell screening, and synthetic library generation, have been pivotal in developing approximately 80 FDA-approved antibody therapeutics. Existing antibody discovery methods have mostly failed for certain target groups, such as G protein-coupled receptors (GPCRs) and ion channels.

FDA Approval 147

FDA Approval Disease FDA Treatment 147

The Pharma Data

JANUARY 20, 2021

FDA Approves Verquvo (vericiguat) for Heart Failure with Reduced Ejection Fraction. When NO binds to sGC, the enzyme catalyzes the synthesis of intracellular cyclic guanosine monophosphate (cGMP), a second messenger that plays a role in the regulation of vascular tone, cardiac contractility, and cardiac remodeling.

FDA Approval 52

FDA Approval FDA Hospitals Treatment 52

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 Discussions As a result of the feedback received in 2019, the FDA outlined the five-pillar approach to regulating AI/ML.

FDA 52

FDA Drug Development Regulations FDA Approval 52

Agency IQ

SEPTEMBER 15, 2023

FDA’s nonprescription advisors find no efficacy for phenylephrine This week, FDA’s Nonprescription Drugs Advisory Committee (NDAC) voted unanimously that current scientific data do not support the efficacy of oral phenylephrine as a nasal decongestant, aligning with FDA analysis — and re-analysis — of data.

Science 59

Science FDA Approval FDA Regulations 59

Drug Discovery World

OCTOBER 18, 2023

We’re beginning to see their potential come to fruition, with the FDA having approved three treatments as of January 2023 — for retinal dystrophy 1 spinal muscular atrophy, 2 and haemophilia B. Food & Drug Administration (2017) FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss.

Therapies 162

Therapies Clinical Trials FDA Approval Virus 162

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.

FDA 59

FDA Regulations FDA Approval Production 59

FDA Law Blog: Biosimilars

JULY 24, 2023

Koblitz — Neither Jazz Pharmaceuticals nor Avadel CNS Pharmaceuticals has taken the battle of sodium oxybate—a drug approved to treat narcolepsy—lying down. After suing each other in patent litigation and Avadel’s suit against FDA challenging the Agency’s authority to compel patent certifications, it’s Jazz’s turn to sue FDA.

FDA 52

FDA Regulations Clinical Trials Production 52

FDA Law Blog: Biosimilars

SEPTEMBER 19, 2023

Industry has long requested input from FDA about the types of patents that can be listed in the Orange Book, and, in response, FDA asked for comments on the types of patents that should be listed. But FDA has never made an affirmative statement as to the types of patents that can be listed in the Orange Book.

FDA 105

FDA Biosimilars FDA Approval Drugs 105

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]

FDA Approval 52

FDA Approval FDA Treatment Pharmaceutical Companies 52

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 determined that food from GalSafe pigs is safe for the general population to eat. SILVER SPRING, Md. ,

FDA Approval 52

FDA Approval FDA Production Vaccine 52

The Pharma Data

AUGUST 21, 2020

The FDA has authorised Novartis’ Kesimpta (ofatumumab) for treating patients with relapsing forms of multiple sclerosis (MS), it has been revealed. . The injectable treatment also covers other forms of MS including clinically isolated syndrome and active secondary progressive disease. It can also be self-administered once a month.

FDA Approval 52

FDA Approval Disease FDA Drugs 52

The Pharma Data

MAY 13, 2021

Third time’s the charm as Heron wins FDA nod for non-opioid anesthetic Zynrelef ( Endpoints ). Amgen, AstraZeneca bolster their case for breakthrough asthma program as FDA considers taking up a review ( Endpoints ). Japan vaccine chief blames drug approval system for slow inoculation drive ( Reuters ).

FDA Approval 52

FDA Approval FDA Vaccine Pharmacy 52

Agency IQ

JUNE 17, 2024

But in a major decision today, the Supreme Court unanimously ruled that The Alliance for Hippocratic Medicine and the doctors it represented had not demonstrated adequate proof of standing to sue the FDA, offering the agency significant relief from the prospect of endless litigation. In the case of Alliance for Hippocratic Medicine v.

FDA 40

FDA Doctors Testimonials Regulations 40

Drug Discovery World

AUGUST 14, 2023

While CGT is still an emerging market, its growth has accelerated over the last five years, resulting in a wave of products approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The EMA, however, doesn’t require the use of an agency-approved diagnostic.

Marketing 130

Marketing DNA Therapies FDA 130

FDA Law Blog: Biosimilars

NOVEMBER 5, 2023

Dr. Won will share his expertise and insights on the intricacies of the United States Food and Drug Administration (FDA) regulations pertaining to medical devices, with a special focus on class II and class III hearing devices. Philip Won , of Hyman, Phelps & McNamara, P.C.

Science 52

Science FDA Approval FDA Regulations 52

The Pharma Data

APRIL 7, 2023

This drug was approved to reduce the risk of preterm birth in women pregnant with one baby who have a history of spontaneous preterm birth. The decision was issued jointly by the FDA Commissioner and Chief Scientist. Based on that review, they have decided to withdraw approval of Makena and generic versions of Makena.

FDA 40

FDA FDA Approval Production Vaccine 40

The Pharma Data

NOVEMBER 23, 2021

Priority Review designation directs overall attention and resources to the evaluation of applications for drugs that, if approved, would be significant improvements in the safety or effectiveness of the treatment, diagnosis or prevention of serious conditions when compared to standard applications. The FDA, an agency within theU.S.

FDA Approval 52

FDA Approval Treatment FDA DNA 52