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Cell and Gene Therapy, Industry

Vertex islet cell therapy gets type 1 diabetics off insulin

June 24, 2024

Via: Pharmaphorum

The new data from Vertex’s phase 1/2 trial of VX-880, presented at the American Diabetes Association (ADA) annual congress in Florida, suggest that the therapy can restore the physiological activity of the insulin-producing islet cells of the pancreas and restore glucose […]


FDA, Regulations

FDA Releases Facility Readiness Guidance

June 21, 2024

Via: Biopharm International

FDA published a final guidance document on June 18, 2024 that provides applicants with information on FDA’s policy for assigning a goal date for a facility’s inspection readiness as certified on Form FDA 356h, submitted as part of an original […]


News

AbbVie breaks new ground for IL-23 drug Skyrizi in IBD

June 20, 2024

Via: Pharmaphorum

Skyrizi (risankizumab) was previously already cleared by the FDA for adults with moderately to severely active Crohn’s disease, active psoriatic arthritis, and moderate to severe plaque psoriasis. The approval in UC is based on the results of a 12-week induction study called […]


Cell and Gene Therapy, FDA, Industry, Regulations

Sarepta Duchenne gene therapy wins broader use from FDA

June 20, 2024

Via: Biopharma Dive

The Food and Drug Administration has substantially loosened limits on the first gene therapy for Duchenne muscular dystrophy in a decision that could greatly expand its use even as questions remain about its effectiveness. The agency on Thursday made the therapy, called […]


Editorial

Understanding the drivers behind the US drug shortage

June 20, 2024

Via: Camila Mendes

Annual drug shortages are common in the US and indeed throughout the world and can be attributed to a few key issues. Experts have pointed out how policy can assist in regulating the production, manufacturing, distribution, and purchase of essential […]


FDA, Regulations

AbbVie’s IL-23 inhibitor Skyrizi approved by FDA to treat ulcerative colitis in adults

June 19, 2024

Via: PMLiVE

Affecting more than one million people in the US, UC is a type of inflammatory bowel disease (IBD) that causes inflammation in the digestive tract and can result in damage to the colon lining. Patients often experience a range of […]


FDA, Regulations

FDA Approves Immunoglobulin Therapeutic to Treat Primary Immunodeficiencies

June 18, 2024

Via: Biopharm International

Global healthcare company, Grifols, announced on June 17, 2024 that its subsidiary Biotest has garnered its first FDA approval with the intravenous immunoglobulin (Ig) therapeutic, Yimmugo, which Grifols hopes to launch commercially in the United States in the second half […]


FDA, Regulations

FDA clears Roche digital pathology platform for diagnosis

June 18, 2024

Via: Pharmaphorum

The clearance covers Roche’s Ventana DP 200 slide scanner, digital pathology workflow software, and a display that can be used to review and interpret digital images of scanned pathology slides. Proponents of digital pathology technologies say that having this image data in […]


News

Study finds low secondary cancer risk with CAR-Ts

June 17, 2024

Via: Pharmaphorum

Last November, the FDA issued a warning about a risk of secondary cancers – particularly T-cell cancers – and earlier this year it requested a change to safety labelling to include a ‘black box’ warning on all six of the […]


FDA, Regulations

Amgen’s Blincyto approved by FDA for new acute lymphoblastic leukaemia indication

June 17, 2024

Via: PMLiVE

The therapy has been authorised for use in patients with CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukaemia (B-ALL) in the consolidation phase, regardless of measurable residual disease (MRD) status. With more than 6,500 new cases diagnosed in the US […]


FDA, Industry, Regulations, Vaccines

FDA Tells Vaccine Makers to Target New COVID Variant for Fall

June 14, 2024

Via: Drugs.com

COVID vaccine makers will be advised to update their shots to target the KP.2 variant, an offshoot of the JN.1 variant that spread widely last winter, the U.S. Food and Drug Administration announced Thursday. It’s a turnaround for the agency: […]


FDA, Regulations

FDA widens label for BMS’ cancer drug Augtyro

June 14, 2024

Via: Pharmaphorum

Augtyro (repotrectinib), which first got a green light from the FDA as a treatment for ROS1-positive non-small-cell lung cancer (NSCLC) last November, has now had its label extended to include all solid tumours that have NTRK gene fusions. The NSCLC approval was […]


Cell and Gene Therapy, Industry

Pfizer setback brings questions for Duchenne gene therapy ahead of Sarepta decision

June 13, 2024

Via: Biopharma Dive

The failure of a Pfizer medicine for Duchenne muscular dystrophy adds new uncertainty around the effectiveness of gene therapy for the muscle-wasting condition, days before the Food and Drug Administration is expected to decide on expanding use of a similar […]


Clinical Trials, Research and Development

EMA announces launch of two advice pilots to improve clinical trials in Europe

June 12, 2024

Via: PMLiVE

The pilots aim to improve the quality of applications for clinical trials and lay the foundation for the development of safe and effective medicines in Europe. First launched in January 2022, the ACT EU initiative seeks to transform how clinical […]


FDA, Regulations

FDA Advisors Support New Alzheimer’s Drug, Donanemab

June 11, 2024

Via: Drugs.com

A U.S. Food and Drug Administration advisory panel voted unanimously on Monday to recommend that the benefits of a new drug for Alzheimer’s outweigh its harms, which can include brain swelling and bleeding. Eli Lilly’s donanemab did slow declines in thinking skills […]


News

Drug Shortage Reports Emphasize Continuing Problems

June 11, 2024

Via: Biopharm International

In early June 2024, FDA and the US Pharmacopeial Convention (USP) both published reports on drug shortages as of the end of 2023. The Center for Drug Evaluation and Research at FDA published its 11th annual report to Congress on […]


FDA, Industry, Regulations, Vaccines

FDA Gives Nod to RSV Vaccine for People in Their 50s

June 10, 2024

Via: Drugs.com

The U.S. Food and Drug Administration on Friday has for the first time approved the use of a respiratory syncytial virus (RSV) vaccine for people in their 50s who are at increased risk for the illness. Drugmaker GSK’s Arexvy vaccine, […]


FDA, Regulations

FDA OKs wider use of Almirall’s actinic keratosis drug

June 10, 2024

Via: Pharmaphorum

First approved in 2020, first-in-class microtubule inhibitor Klisyri (tirbanibulin 1%) reached the market in early 2021, joining a short list of therapies in the US that are available for AK, which is a major public health concern as it can […]


FDA, Regulations

With Bird Flu a Threat, FDA Asks Some States to Curb Sales of Raw Milk

June 7, 2024

Via: Drugs.com

The U.S. Food and Drug Administration on Thursday asked states to work harder to protect the public from the risks of raw milk as a bird flu outbreak continues to spread among dairy cows. In an open letter to state […]


FDA, Regulations

FDA Rescinds Ban on Juul E-Cigarettes

June 7, 2024

Via: Drugs.com

A ban on Juul e-cigarettes has been reversed, the U.S. Food and Drug Administration announced Thursday. Why? The agency said it needs to review both new court decisions and updated data from the vape maker. While the company’s e-cigarettes are […]