Advarra

AUGUST 16, 2022

In 2016, the FDA granted 333 (57%) of the 582 orphan drug designation requests it received – nearly 10 times the number of designations the agency awarded in 1986. The agency’s 2019 guidance, Rare Diseases: Common Issues in Drug Development , called attention to the need for natural history studies for rare diseases.

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Drug Target Review

JULY 10, 2023

This deeper understanding has enabled the development of numerous potentially breakthrough therapies, which are currently being tested in the clinic. As scientists continue to elucidate and advance novel drug targets and technology applications, the goal of bringing impactful treatments to patients becomes more of a reality.

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The Pharma Data

JUNE 25, 2021

Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for lecanemab (BAN2401), an investigational anti-amyloid beta (A?) Breakthrough Therapy designation is an FDA program intended to expedite the development and review of medicines for serious or life-threatening conditions.

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Agency IQ

AUGUST 16, 2024

By Amanda Conti | Aug 13, 2024 10:00 PM CDT Regulatory context: Psychedelic regulation and drug development A growing body of evidence suggests that psychedelics may provide clinical benefit for certain purposes, especially mental health conditions. Importantly, midomafetamine was proposed for use in conjunction with psychotherapy.

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Alta Sciences

JUNE 27, 2023

They remind us of the immense benefits of drug development. For Michelle Newby, Study Coordinator II for preclinical services at Altasciences, research strikes a personal chord: In 2017, at the age of 25, Michelle was diagnosed with cancer. A: In December of 2017, I was diagnosed with two different types of stage III leukemia.

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The Pharma Data

OCTOBER 21, 2020

In 2017, the most recent year for data in the U.S., Roughly one in three people initially diagnosed in the early stages of the disease go on to develop recurrent advanced or metastatic breast cancer. Daiichi Sankyo is not only partnering with advocacy groups, but is also developing treatments for this common disease.

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The Pharma Data

OCTOBER 26, 2020

(Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process.

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Drug Target Review

OCTOBER 24, 2023

We also believe that drug development teams should routinely consider the potential modifying effect of the gut microbiome on drug potency and factor it into assessments of PK and PD. MB097, our drug with immune-oncology applications, is a consortium of nine bacterial strains. difficile and C. albicans.

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The Pharma Data

DECEMBER 8, 2020

Since partnering with Eli Lilly to produce the first monoclonal antibody therapy approved for mild-to-moderate COVID-19 patients, antibody discovery company AbCellera is going big on the Nasdaq. Tackle the toughest problems in drug development.” The promise to partners is to “move quickly. Reduce cost. Silverback Therapeutics

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Alta Sciences

APRIL 17, 2024

Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.

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Agency IQ

AUGUST 2, 2024

September 30, 2024 PDUFA Novel Approaches to Development of Cell and Gene Therapy Convene a public meeting to solicit input on methods and approaches (e.g., use of RWE, registries) for capturing post-approval safety and efficacy data for cell and gene therapy products.

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The Pharma Data

AUGUST 21, 2020

Eli Lilly, on the heels of detailing a clinical win in China for PD-1 inhibitor Tyvyt, has decided to take a bigger stake in the drug developed by partner Innovent Biologics in an expanded deal that could vault it into the lucrative—yet ultra-competitive—U.S. lung cancer market. approval, potentially speeding time to market.”.

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The Pharma Data

DECEMBER 9, 2020

333-222268) previously filed with the Securities and Exchange Commission (the “SEC”) on December 22, 2017 and declared effective by the SEC on January 5, 2018. Oncternal focuses drug development on promising yet untapped biological pathways implicated in cancer generation or progression. SAN DIEGO, Dec.

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NIH Director's Blog: Drug Development

DECEMBER 4, 2018

This blood vessel-tissue interface can prevent certain molecules—including some potentially beneficial therapies—from entering the brain. It included a chip that models the blood-brain barrier. I’ll be anticipating some some really cool science results in the coming months!

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The Pharma Data

JANUARY 31, 2021

Trimer-Tag© is an innovative drug development platform which allows the production of novel, covalently-trimerized fusion proteins. Dynavax is a commercial stage biopharmaceutical company developing and commercializing novel vaccines. CEPI has initiated 11 partnerships to develop vaccines against the novel coronavirus.

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NIH Director's Blog: Drug Development

JULY 20, 2017

Helping to fuel this optimism are preliminary results released this week from early-stage clinical trials of three next-generation, triple combination therapies that modulate the function of CFTR. Developing drugs that could rescue the function of mutant CFTR has been a long and difficult journey. 1989 Sep 8;245(4922):1059-1065.

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A Dose of Drug Development

SEPTEMBER 17, 2018

How Integrium as a company plays a role in advanced technology such as these is that Integrium has the advantage of having 20 years of experience with variety of therapies within its portfolio. Though 2017 may have had more IPOs in 9 months of the year, but the capital raise is considerably different.

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Vial

DECEMBER 11, 2023

According to a study by the Tufts Center for the Study of Drug Development, the average time from site identification to study start-up completion is approximately 31.4 Starting up a new site can be a challenging and time-consuming process, often filled with unforeseen hurdles and delays. weeks for Phase II and Phase III studies.

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Advarra

AUGUST 10, 2023

Rapid growth in gene therapy is expected to receive additional support as the Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) prepares to launch Operation Warp Speed for Rare Diseases. Peter Marks, head of FDA’s CBER – the organization responsible for regulating gene therapies.

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The Pharma Data

DECEMBER 3, 2020

Trimer-Tag © is an innovative drug development platform which allows the production of novel, covalently-trimerized fusion proteins. Additionally, Clover is leveraging its in-house GMP biomanufacturing capabilities to support large-scale production of its biologic therapies. S-Trimer is intended to be adjuvanted. About CEPI.

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The Pharma Data

OCTOBER 29, 2020

If approved, aducanumab would become the first therapy to reduce the clinical decline of Alzheimer’s disease and to meaningfully change the course of Alzheimer’s disease. If approved, aducanumab would become the first therapy to reduce the clinical decline in patients with Alzheimer’s disease. CAMBRIDGE, Mass.

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The Pharma Data

JULY 29, 2021

today announced results of a longitudinal preliminary assessment of the clinical effects of lecanemab (development code: BAN2401) — granted Breakthrough Therapy designation by the U.S. and slowed clinical decline in an 18-month Phase 2b proof of concept study (Study 201, n=856) in early AD ( Alz Res Therapy 13; 2021 ).

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The Pharma Data

DECEMBER 9, 2020

(Tokyo, Japan) announced that Biogen has submitted a Japanese New Drug Application (J-NDA) to the Ministry of Health, Labor and Welfare (MHLW) for aducanumab, an investigational therapy for Alzheimer’s disease. Biogen licensed aducanumab from Neurimmune under a collaborative development and license agreement.

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The Pharma Data

JANUARY 31, 2021

Trimer-Tag© is an innovative drug development platform which allows the production of novel, covalently-trimerized fusion proteins. CEPI is an innovative partnership between public, private, philanthropic, and civil organisations, launched at Davos in 2017, to develop vaccines to stop future epidemics. About CpG 1018.

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Broad Institute

OCTOBER 11, 2023

But immunotherapy was not yet widely used and had not been applied clinically to Merkel cell carcinoma, so she received traditional chemotherapy and radiation therapy, suffering life-threatening complications along the way. His mother had a presentation of the disease that suggested her immune system was already on the job.

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New Drug Approvals

SEPTEMBER 13, 2024

4] [6] Aticaprant was originally developed by Eli Lilly , was under development by Cerecor for a time, and is now under development by Janssen Pharmaceuticals. [2] 12] Positron emission tomography imaging revealed that brain KORs were almost completely saturated by the drug 2.5 Dhir A (January 2017). August 2017.

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Agency IQ

JULY 8, 2024

The information below includes the event type and whether the meeting can be attended by the public (Open) or not (Closed).

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The Pharma Data

NOVEMBER 6, 2020

Biogen licensed aducanumab from Neurimmune under a collaborative development and license agreement. Since October 2017 Biogen and Eisai have collaborated on the development and commercialization of aducanumab globally. About Aducanumab’s Clinical Program. About Biogen.

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Drug Target Review

SEPTEMBER 26, 2023

Early detection of neurotoxicity induced by potential new therapies is a major challenge, and hiPSC-neuronal cells may provide a solution. CNS-related issues account for nearly a quarter of failures during clinical development, a phase where consequences are high in terms of resources and patient impact. 2017 Jan;155(1):234–47.

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Vial

DECEMBER 5, 2023

It has become a fundamental tool for researchers to explore the complexities of genetic information and conduct genetic-informed drug development. Sponsors have used NGS to screen patients for clinical trial eligibility and patient stratification , expanded CDx development, and comprehensive genomic profiling (CGP).

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Drug Target Review

JUNE 21, 2023

Enabling the systematic discovery of these largely uncharted targets can be a valuable opportunity for development of novel therapies. Genetic-based therapeutics hold significant promise in the clinic Despite the growing number of genes linked to human disease, the proportion of genes targeted by approved therapies remains low.

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Drug Target Review

MAY 23, 2023

Therapies developed in recent decades have transformed the treatment of IBD, making hospitalisation and surgery less common. However, many patients respond poorly to corticosteroid treatment, or their immune system responds unfavourably to biological therapies, such as the development of autoimmune diseases.

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Agency IQ

AUGUST 16, 2024

As opposed to the linear dose-response relationships of chemotherapies, the wide therapeutic indexes of many of these newer therapies means that doses can be increased without simultaneously increasing toxicities. Other issues also arise: Unlike conventional cytotoxic drugs where the relationship between early (e.g.,

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Agency IQ

JULY 12, 2024

The examples : 1) a health care provider posting on the internet about unsubstantiated claims that a class of cholesterol drugs causes Alzheimer’s; 2) a celebrity influencer recommending off-label use (i.e.,

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Agency IQ

FEBRUARY 23, 2024

Workshop addresses oncology dose optimization across full span of development In a series of broad-ranging, frank discussions, attendees at a joint FDA-American Association of Cancer Research (AACR) workshop worked through opportunities and challenges for dose optimization across the span of cancer drug development activities.

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Agency IQ

FEBRUARY 15, 2024

We have tried to sort guidance documents by topic area. If you’re looking for something specific, we recommend using the search function in your browser (Ctrl-F) to look for keywords of interest.

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The Pharma Data

NOVEMBER 15, 2020

This prestigious group of professionals will help lead the Company as it prepares for potential commercialization of its investigational cell therapy, MultiStem ®. Most recently, she was a senior executive at Celgene Corporation, where she led corporate strategy from 2012 to 2017. drug development initiative from 2012 to February 2019.

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