Helixgate

Helixgate

Uncategorized

STAT+: FDA to reconsider treatment for rare cancer after its surprise rejection

Two companies developing a therapy for a rare blood cancer have reached an agreement with the Food and Drug Administration that walked back the agency’s main reason for rejecting the drug in January. 

Pierre Fabre Pharmaceuticals and Atara Biotherapeutics, makers of the drug called Ebvallo, said Thursday that a meeting held in late April with FDA officials ended with the agency agreeing that their already completed, single-arm clinical trial was sufficient to support a review and potential approval. 

When the FDA rejected Ebvallo, the agency said the same study was flawed and the data produced from it was “insufficient” to support the drug’s approval. The drug’s review was conducted by the FDA’s Center for Biologics Evaluation and Research, led at the time by Vinay Prasad. He departed the agency at the end of April. 

Continue to STAT+ to read the full story…

Read More

Published

on

Two companies developing a therapy for a rare blood cancer have reached an agreement with the Food and Drug Administration that walked back the agency’s main reason for rejecting the drug in January. 

Pierre Fabre Pharmaceuticals and Atara Biotherapeutics, makers of the drug called Ebvallo, said Thursday that a meeting held in late April with FDA officials ended with the agency agreeing that their already completed, single-arm clinical trial was sufficient to support a review and potential approval. 

When the FDA rejected Ebvallo, the agency said the same study was flawed and the data produced from it was “insufficient” to support the drug’s approval. The drug’s review was conducted by the FDA’s Center for Biologics Evaluation and Research, led at the time by Vinay Prasad. He departed the agency at the end of April. 

Continue to STAT+ to read the full story…

Read More

Continue Reading
Click to comment

Leave a Reply

Your email address will not be published. Required fields are marked *

Uncategorized

STAT+: Pharmalittle: We’re reading about Sanofi and an FDA voucher, FDA rethinking a rejection, and more

Top of the morning to you, and a fine one it is. Sunny skies and mild breezes are enveloping the Pharmalot campus once again. And to celebrate, we are brewing still more cups of stimulation and inviting you to join us. Our choice today is Jack Daniel’s. Yes, this is a real thing. And remember, a prescription is not required. So no need to mess with rebates, coupons, or TrumpRx. Meanwhile, here are a few items of interest. Hope you have a smashing day and conquer the world. And of course, do stay in touch. We appreciate feedback, criticism and tips. …

Sanofi asked the U.S. Food and Drug Administration to pull its type 1 diabetes drug, teplizumab, out of FDA Commissioner Marty Makary’s new speedy review program, STAT reports. The move comes after acting Center for Drug Evaluation and Research Director Tracy Beth Høeg disagreed with a staff decision to approve the drug. The agency missed its April 21 goal date to deliver a decision to Sanofi. Such decisions are typically made by career scientists. It is rare for a center director to become involved in scientific review of a single drug, and particularly a political appointee like Høeg. Makary recently said that he stands behind review teams, and that “disaster” occurs whenever political leaders overrule scientific staff. 

An experimental drug from Revolution Medicines that nearly ​doubled survival time for patients with advanced pancreas cancer in clinical trials comes with a high rate ‌of mostly low-grade side effects, Reuters tells us. A report by researchers from a first-in-human trial of daraxonrasib is the first peer-reviewed paper to show safety data for what analysts say could become the next standard of care for previously treated metastatic pancreatic cancer. Among the 168 patients with previously treated pancreatic ductal adenocarcinoma who received daraxonrasib in the early trial, treatment-related adverse side effects ​of any grade occurred in 96%, while severe or life-threatening events were reported in 30%.

Continue to STAT+ to read the full story…

Read More

Published

on

Top of the morning to you, and a fine one it is. Sunny skies and mild breezes are enveloping the Pharmalot campus once again. And to celebrate, we are brewing still more cups of stimulation and inviting you to join us. Our choice today is Jack Daniel’s. Yes, this is a real thing. And remember, a prescription is not required. So no need to mess with rebates, coupons, or TrumpRx. Meanwhile, here are a few items of interest. Hope you have a smashing day and conquer the world. And of course, do stay in touch. We appreciate feedback, criticism and tips. …

Sanofi asked the U.S. Food and Drug Administration to pull its type 1 diabetes drug, teplizumab, out of FDA Commissioner Marty Makary’s new speedy review program, STAT reports. The move comes after acting Center for Drug Evaluation and Research Director Tracy Beth Høeg disagreed with a staff decision to approve the drug. The agency missed its April 21 goal date to deliver a decision to Sanofi. Such decisions are typically made by career scientists. It is rare for a center director to become involved in scientific review of a single drug, and particularly a political appointee like Høeg. Makary recently said that he stands behind review teams, and that “disaster” occurs whenever political leaders overrule scientific staff. 

An experimental drug from Revolution Medicines that nearly ​doubled survival time for patients with advanced pancreas cancer in clinical trials comes with a high rate ‌of mostly low-grade side effects, Reuters tells us. A report by researchers from a first-in-human trial of daraxonrasib is the first peer-reviewed paper to show safety data for what analysts say could become the next standard of care for previously treated metastatic pancreatic cancer. Among the 168 patients with previously treated pancreatic ductal adenocarcinoma who received daraxonrasib in the early trial, treatment-related adverse side effects ​of any grade occurred in 96%, while severe or life-threatening events were reported in 30%.

Continue to STAT+ to read the full story…

Read More

Continue Reading

Uncategorized

Entrada crashes as Duchenne therapy comes in ‘below expectations’ in early study

Published

on

Entrada Therapeutics saw a 2.36% dystrophin increase in patients treated with its oligonucleotide asset—a number that fell short of the company’s prior guidance of a double-digit improvement.

Continue Reading

Uncategorized

Next Gen Leadership Awards Presented at the AGBT Agricultural Meeting

Published

on

Last month, the AGBT Agricultural Meeting was held in Phoenix, Arizona. The conference is focused on agricultural genomics—plant and animal genetics. During the meeting, the recipients of the 2026 Next Gen Leadership Awards were announced.

These awards recognize outstanding early-career scientists and graduate students whose work and potential are shaping the future of agricultural genomics, including advances in plant and animal genomics. Award recipients receive financial support to attend and present their research at the AGBT Agricultural Meeting, with opportunities to engage with leaders in the field and build connections across the genomics community.

“These awardees reflect the strength and diversity of emerging talent in agricultural genomics,” said Sarah Hearne, PhD, chief science and innovation officer at CIMMYT and co-chair of the AGBT Agriculture Scientific Organizing Committee. “AGBT Agriculture plays an important role in bringing these scientists into conversation with leaders across the field, helping accelerate the translation of genomics into practice.”

The awardees represent rising leaders in agricultural genomics, advancing research across genomic variability, genetic analysis, molecular diagnostics, pathogen surveillance, and quantitative trait genomics to improve crop performance, strengthen food safety, and advance sustainable agriculture.

“This award represents a transformative opportunity to grow as a scientist and contribute more effectively to innovation in animal breeding,” said Larissa Bordin Temp, a 2026 Next Gen Leadership Award recipient.

The 2026 AGBT Agricultural Meeting Next Gen Leadership awardees were:

  • Boris ME Alladassi, PhD: postdoctoral research associate at the University of Illinois Urbana-Champaign
    • Research focus: Connecting the evolutionary and statistical views of epistasis in quantitative trait genomics
  • Mythri Bikkasani: graduate student at Punjab Agricultural University, India
    • Research focus: Connecting the dots: from high-throughput feed phenotyping to genomic dissection of heterosis in maize
  • Larissa Bordin Temp: graduate student at São Paulo State University, Faculty of Agricultural and Veterinary Sciences
    • Research focus: Genomic evaluation of rump fat–adjusted residual feed intake in zebu cattle: implications for selection strategies
  • Lauren Johnson: graduate student at Gluck Equine Research Center, University of Kentucky
    • Research focus: Functional introgression within the horse mhc genes
  • Mehak Kapoor: graduate assistant at Iowa State University
    • Research focus: Cell-type resolved gene expression signatures to identify and predict persistent PRRSV infection
  • Pedro Nuñez Romano, PhD: postdoctoral researcher at Universitat Politècnica de València
    • Research focus: Integrating technology to refine the estimation of social genetic effects in pigs
  • Viona Osei: graduate student at Tuskegee University
    • Research focus: Exploiting genomic variability in Listeria for the development of molecular diagnostic markers
  • Kyungyong Seong, PhD: postdoctoral fellow at the University of California, Davis
    • Research focus: Resurrection of the plant immune receptor Sr50 to overcome pathogen immune evasion
  • Jade van Wijk: graduate student at Earlham Institute
    • Research focus: Using airborne DNA sequencing to monitor sporulation, infection and relative abundance of cereal rust fungi

The post Next Gen Leadership Awards Presented at the AGBT Agricultural Meeting appeared first on GEN – Genetic Engineering and Biotechnology News.

Continue Reading
Advertisement

Trending