Development and Regulation - Clinical Research Informer

FDA rejects two China-developed cancer drugs

Bio Pharma Dive

MAY 2, 2022

The regulator cited concerns around single-country trials in turning back Hutchmed's pancreatic cancer treatment, while manufacturing issues held up Junshi and Coherus' throat cancer medicine.

Development

Development Regulation Manufacturing Drugs

C4XD and AstraZeneca enter $402m deal to develop respiratory disease therapy

Pharmaceutical Technology

NOVEMBER 28, 2022

C4X Discovery (C4XD) and AstraZeneca have entered an exclusive global licensing agreement worth up to $402m to develop oral therapy to treat inflammatory and respiratory ailments. The deal has been signed to develop the C4X NRF2 Activator programme for these ailments. This includes an upfront payment of $2m from AstraZeneca.

Development

Development Gene Expression Licensing Licensing

FDA’s lab-developed test rule could be first test of agency’s power post-Chevron

Bio Pharma Dive

JULY 11, 2024

The Supreme Court’s decision to overturn the Chevron doctrine would make it easier to challenge agency regulations, such as the LDT final rule.

Regulation

Regulation Development

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Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

FDA Law Blog

NOVEMBER 10, 2024

Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.

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Astellas to support development of Taysha’s gene therapy programmes

Pharmaceutical Technology

OCTOBER 25, 2022

Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). This would allow the companies to boost the development of new therapy options for Rett syndrome and GAN patients.

Gene Therapy

Gene Therapy Gene Development Genetics

Gatehouse Bio and AstraZeneca to develop RNA therapeutics for heart failure

Pharmaceutical Technology

SEPTEMBER 9, 2022

US-based biotech company Gatehouse Bio is extending its collaboration with AstraZeneca for developing RNA therapeutics to treat heart failure with preserved ejection fraction (HFpEF). It is expected to expedite the development of a wide range of new therapeutics using the small non-coding area of the genome that is not explored.

RNA

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FDA clears Rocket to resume testing gene therapy for a rare heart disease

Bio Pharma Dive

AUGUST 16, 2021

The decision from the regulator ends a three-month study pause that had stalled Rocket's development of of the Danon disease treatment.

Gene Therapy

Gene Therapy Gene Regulation Development

The Benefits of Integrated CDMO Services: Streamlining Drug Development and Manufacturing

Drug Patent Watch

DECEMBER 10, 2024

The pharmaceutical industry has undergone significant changes over the past decade, with a growing trend towards outsourcing key aspects of research, development, and manufacturing to third-party vendors. The Rise of Integrated CDMOs The global biotechnology and pharmaceutical services outsourcing market size was valued at $70.48

Manufacturing

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In the News: August 2021 Regulatory and Development Updates

Camargo

SEPTEMBER 8, 2021

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. devices must be regulated as devices, and drugs—if they do not also satisfy the device definition—must be regulated as drugs.”.

Development

Development Regulation Production Drugs

Rivus raises $132m to advance clinical development of obesity treatment

Pharmaceutical Technology

SEPTEMBER 23, 2022

The CMAs of the company can lower fat throughout the body through mitochondrial uncoupling, a natural process that causes energy regulation. The post Rivus raises $132m to advance clinical development of obesity treatment appeared first on Pharmaceutical Technology.

Clinical Development

Clinical Development Development Life Science Trials

Judge overturns FDA’s lab developed test regulation, siding with industry

Bio Pharma Dive

APRIL 1, 2025

A Texas judge vacated the FDA’s final rule, which was strongly opposed by the laboratory industry, and remanded the matter to HHS Secretary Robert F. Kennedy Jr.

Regulation

Regulation Development

AbbVie keeps up dealmaking pace in neuroscience

Bio Pharma Dive

MARCH 11, 2022

On the heels of its Syndesi acquisition, the company has announced an agreement with Gedeon Richter focused on researching and developing new dopamine-regulating drugs for neuropsychiatric diseases.

Regulation

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In the News: October Regulatory and Development Updates

Camargo

NOVEMBER 11, 2020

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. Labeling regulations prohibit reminder ads for products with boxed warnings, which the FDA requires when a drug product has very serious risks.

Development

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PCIM&H soon to develop pharmacopoeia standards for Ayurveda, Siddha, Unani & Homoeopathy drugs

AuroBlog - Aurous Healthcare Clinical Trials blog

APRIL 4, 2023

The Pharmacopoeia Commission of Indian Medicine and Homoeopathy (PCIM&H) may soon undertake development of pharmacopoeia standards for the Ayurveda, Siddha, Unani and Homoeopathy drugs under the ‘One Herb One Standard’ (OHOS) among other initiatives during the financial year 2023-24.

Development

Development Drugs Regulation Medicine

Regulators develop five principles for machine learning-enabled medical devices

Pharma Times

OCTOBER 27, 2023

The new principles will remove the regulatory burden for MLMD manufacturers - News - PharmaTimes

Regulation

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Asceneuron raises another $100M for Alzheimer’s drug development

Bio Pharma Dive

JULY 16, 2024

The Merck KGaA spinout has a tau-regulating medicine it claims could be an “ideal therapy” for Alzheimer’s. The pitch has intrigued both healthcare investors and pharma venture arms.

Development

Development Regulation Drugs Medicine

MHRA revamps UK clinical trial regulation with the promise of faster timelines

Pharmaceutical Technology

MARCH 23, 2023

This week, the Medicines and Healthcare products Regulatory Agency (MHRA) is introducing major changes to clinical trial regulation in the UK with expedited timelines at several stages. The MHRA hopes this new framework “will remove obstacles to innovation” and “streamline the regulation of clinical trials” amongst other things.

Clinical Trials

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Innovative Approaches to Generic Drug Development: Case Studies

Drug Patent Watch

JANUARY 14, 2025

But how do companies develop these cost-effective alternatives? Generic drug manufacturers are taking a similar approach by developing multiple drugs simultaneously[1]. ”[2] This study not only addressed the ATA’s concerns but also highlighted the power of real-world evidence in generic drug development and regulation.

Generic Drugs

Generic Drugs Development Drugs Manufacturing

Bristol Myers to buy KRAS cancer drug developer Mirati for $4.8B

Bio Pharma Dive

OCTOBER 8, 2023

Mirati shareholders could also receive another $1 billion should an experimental cancer drug the biotech is developing make it to U.S. regulators’ desks.

Development

Development Drugs Regulation

Roche to acquire biopharma firm Good Therapeutics for $250m

Pharmaceutical Technology

SEPTEMBER 8, 2022

Good Therapeutics focuses on the development of PD-1-regulated IL-2 drugs that are based on innovative conditionally active drug technology. With the takeover, Roche will attain rights to a conditionally active, PD-1-regulated IL-2 programme of Good Therapeutics.

Regulation

Regulation Development Manufacturing Drugs

Compliance Challenges in Generic Drug Development: Lessons Learned

Drug Patent Watch

MARCH 26, 2025

The Unseen Hurdles of Generic Drug Development: Lessons Learned As the pharmaceutical industry continues to evolve, one thing remains constant: the pursuit of affordable, high-quality medications for patients worldwide. But what can be done to mitigate these risks and ensure the success of generic drug development?

Generic Drugs

Generic Drugs Compliance Development Drugs

Risk Management Strategies for Generic Drug Development

Drug Patent Watch

JANUARY 21, 2025

Navigating the Complex World of Generic Drug Development: Risk Management Strategies to Know As a generic drug developer, you're no stranger to the challenges of bringing affordable medications to market. One of the biggest risks generic drug developers face is patent infringement. But patent risk is just the tip of the iceberg.

Generic Drugs

Generic Drugs Development Drugs Regulation

The opportunities and challenges facing rare disease therapies developers

pharmaphorum

FEBRUARY 23, 2024

Explore the opportunities and challenges facing rare disease therapies developers, including the impact of Rare Disease Day, FDA regulations, and cutting-edge technologies like CRISPR gene therapy.

Gene Therapy

Gene Therapy Development Gene Regulation

Three regulators accept Bristol Myers’ applications for myeloma therapy

Pharmaceutical Technology

APRIL 18, 2023

Three regulators in different jurisdictions have accepted Bristol Myers Squibb’s applications for Abecma (idecabtagene vicleucel) for earlier use to treat triple-class exposed relapsed and/or refractory multiple myeloma in adult patients.

Regulation

Regulation Gene Therapy Gene Development

Interview: Is global AI regulation really revolutionizing pharma and clinical trials?

Outsourcing Pharma

APRIL 29, 2024

OSP spoke to Berkeley Research Group healthcare managing director, Wendy Cheng for a discussion around developments in AI regulation for pharmaceuticals, clinical trials, and drug development at a global scale.

Clinical Trials

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In the News: October 2021 Regulatory and Development Updates

Camargo

NOVEMBER 15, 2021

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. While not fatal to the application, this development is certainly a setback for the sponsor, primarily in terms of a delay in approval.

Development

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Biotech execs say IRA is causing unintended effects for cancer drug development

Pharmaceutical Technology

FEBRUARY 28, 2024

IRA regulations have changed how pharma is approaching innovation in cancer drug discovery and development.

Development

Development Drugs Regulation

Kite and Epic Bio partner to develop new cancer cell therapies

Pharmaceutical Technology

NOVEMBER 1, 2023

Kite has signed agreement with Epic Bio for the development of new cancer cell therapies leveraging the latter’s gene regulation platform.

Development

Development Gene Regulation

In the News: September Regulatory and Development Updates

Camargo

OCTOBER 14, 2020

Each month, Camargo’s “In the News” series highlights important changes and advancements in the regulatory and development space and explores how those changes could impact your program. The FDA has also recently launched the Software Pre-Certification Pilot Program to provide an efficient regulatory framework for developers of SaMD products.

Development

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PDUFA VII: Implications for Developers of Cell and Gene Therapies

Camargo

DECEMBER 20, 2021

One of the biopharmaceutical industry’s most closely watched forthcoming regulatory developments is the FDA’s imminent reauthorization of the Prescription Drug User Fee Act (PDUFA). The increase is designed to strengthen CBER’s capacity and capabilities for regulating cell and gene therapies. 2024: 48 staffers. 2025: 29 staffers.

Gene Therapy

Gene Therapy Gene Development Production

NMDA Regulator by Boehringer Ingelheim International for Unspecified Central Nervous System Disorders: Likelihood of Approval

Pharmaceutical Technology

JANUARY 5, 2025

NMDA Regulator is under clinical development by Boehringer Ingelheim International and currently in Phase I for Unspecified Central Nervous System Disorders.

Regulation

Regulation Clinical Development Development

Biologic Therapeutics Development, Part 2: Regulatory Pathways and Pharmacometric Analysis

Camargo

DECEMBER 13, 2021

The development of biological products (or biologics) represents a major advancement in modern medicine, enabling the treatment of patients with many illnesses where no other therapeutics were previously available. The regulations regarding BLAs for therapeutic biological products are included in 21 CFR parts 600 , 601 , and 610.

Development

Development Production Immune Response Licensing

The Importance of Pharmacovigilance in Generic Drug Development

Drug Patent Watch

MARCH 4, 2025

The Unseen Heroes of Generic Drug Development: Pharmacovigilance As a generic drug developer, you're no stranger to the challenges of bringing affordable medications to market. But have you ever stopped to think about the unsung heroes of generic drug development? Share your experiences and insights in the comments below!

Generic Drugs

Generic Drugs Development Drugs Production

How competitors are collaborating to shape biopharma best practices and regulations of the future

Pharmaceutical Technology

DECEMBER 20, 2022

From partnerships to open-source technology and data transparency, the culture behind therapeutic development and delivery is shifting towards one that embraces teamwork. The collaborative spirit arising between pharmaceutical and biotechnology companies is leading to a new rapport with regulators, as well. Moving forward.

Regulation

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Single-Arm Trials Using Real-World Evidence for Rare Disease Product Development

Camargo

OCTOBER 15, 2020

Similarly, in rare disease drug development, where a placebo control group may not be an option, a single-arm trial can be a successful approach. Natural history data becomes critical for product development when there is no concurrent control. For more insights on RWE, see the Camargo Blog and the FDA’s framework for RWE program.

Production

Production Trials Development Clinical Trials

Logistics in Focus: Orchestrating the Challenges of Cell Therapy

Worldwide Clinical Trials

NOVEMBER 12, 2024

Despite their exciting potential, the smooth operation of cell therapy development trials requires extraordinary orchestration, perfectly aligning the product and patient journeys. The process, from patient coordination through manufacturing and administration, is intricate, time-sensitive, and highly regulated.

Gene Therapy

Gene Therapy Clinical Supply Trials Gene

World Orphan Drug Congresses Europe vs. The US: Global Perspectives on Genetic Testing

Worldwide Clinical Trials

DECEMBER 19, 2024

As regulatory frameworks like the General Data Protection Regulation (GDPR) have heightened the emphasis on data privacy and individual rights, Europe is leading the way in terms of privacy and data. In-Vitro Diagnostic Regulation and The FDAs Final Rule The WODC U.S.

Genetics

Genetics In-Vitro Drugs Clinical Trials

Understanding the Regulatory Environment in Japan for Generic Drug Development

Drug Patent Watch

DECEMBER 30, 2024

The regulatory environment in Japan for generic drug development is complex and has undergone significant changes in recent years. Generic Drug Development To market a generic drug in Japan, an ANDA must be submitted to the PMDA. Conclusion The regulatory environment in Japan for generic drug development is complex and evolving.

Generic Drugs

Generic Drugs Bioequivalency Development Drugs

Radiopharmaceutical deals drive sector growth despite supply crisis

Pharmaceutical Technology

FEBRUARY 4, 2025

Developers celebrate gains for advances with radiopharmaceuticals while regulators and isotope suppliers work to keep up.

Regulation

Regulation Development

BioMarin reports cancer case in hemophilia gene therapy trial

Bio Pharma Dive

SEPTEMBER 13, 2022

The development comes three weeks after Roctavian was approved in Europe and ahead of a planned regulatory submission in the U.S. Drug regulators have not ordered a trial hold, however.

Gene Therapy

Gene Therapy Trials Gene Regulation

MEI and Kyowa Kirin to discontinue development of zandelisib outside Japan

Pharmaceutical Technology

DECEMBER 6, 2022

MEI Pharma and Kyowa Kirin have announced plans to discontinue the international development of investigational cancer therapy, zandelisib, for B-cell malignancies outside of Japan. The latest development comes after the companies received guidance from a meeting held with the US Food and Drug Administration (FDA) in November.

Development

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Cannabinoids receptors: popular preclinical target but banned in 137 countries

Pharmaceutical Technology

DECEMBER 5, 2022

In 2022, a surge in the pipeline has led to cannabinoid receptors becoming the most popular target in preclinical development. Collectively, cannabinoid receptors (CB1 and CB2) are currently the most popular targets in preclinical stage of development, with 391 drugs tagged in total.

Regulation

Regulation Drugs Development Marketing

Move to relax clinical trial norms will cut Indian pharma’s human studies cost, lower drug prices for patients

AuroBlog - Aurous Healthcare Clinical Trials blog

AUGUST 25, 2024

The recent regulatory developments to relax clinical trial norms in India are poised to have a significant impact on the pharmaceutical industry and drug pricing. By easing regulations, Indian pharma could see a reduction in the costs associated with human studies.

Clinical Trials

Clinical Trials Clinical Trials Trials Drugs

Study finds the metabolic regulator MYC is defective in obesity

Medical Xpress

APRIL 27, 2023

The latest findings are of critical importance to the study of MAIT cells and the development of metabolic disease such as obesity. Research led by Maynooth University and published today in the journal Science Signaling has found that the protein MYC is essential for MAIT cell growth, proliferation, metabolism and function.

Regulation

Regulation Protein Development Research

FDA rejects two China-developed cancer drugs

C4XD and AstraZeneca enter $402m deal to develop respiratory disease therapy

Webinars

Trending Sources

FDA’s lab-developed test rule could be first test of agency’s power post-Chevron

Webinars

Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

Astellas to support development of Taysha’s gene therapy programmes

Gatehouse Bio and AstraZeneca to develop RNA therapeutics for heart failure

FDA clears Rocket to resume testing gene therapy for a rare heart disease

The Benefits of Integrated CDMO Services: Streamlining Drug Development and Manufacturing

In the News: August 2021 Regulatory and Development Updates

Rivus raises $132m to advance clinical development of obesity treatment

Judge overturns FDA’s lab developed test regulation, siding with industry

AbbVie keeps up dealmaking pace in neuroscience

In the News: October Regulatory and Development Updates

PCIM&H soon to develop pharmacopoeia standards for Ayurveda, Siddha, Unani & Homoeopathy drugs

Regulators develop five principles for machine learning-enabled medical devices

Asceneuron raises another $100M for Alzheimer’s drug development

MHRA revamps UK clinical trial regulation with the promise of faster timelines

Innovative Approaches to Generic Drug Development: Case Studies

Bristol Myers to buy KRAS cancer drug developer Mirati for $4.8B

Roche to acquire biopharma firm Good Therapeutics for $250m

Compliance Challenges in Generic Drug Development: Lessons Learned

Risk Management Strategies for Generic Drug Development

The opportunities and challenges facing rare disease therapies developers

Three regulators accept Bristol Myers’ applications for myeloma therapy

Interview: Is global AI regulation really revolutionizing pharma and clinical trials?

In the News: October 2021 Regulatory and Development Updates

Biotech execs say IRA is causing unintended effects for cancer drug development

Kite and Epic Bio partner to develop new cancer cell therapies

In the News: September Regulatory and Development Updates

PDUFA VII: Implications for Developers of Cell and Gene Therapies

NMDA Regulator by Boehringer Ingelheim International for Unspecified Central Nervous System Disorders: Likelihood of Approval

Biologic Therapeutics Development, Part 2: Regulatory Pathways and Pharmacometric Analysis

The Importance of Pharmacovigilance in Generic Drug Development

How competitors are collaborating to shape biopharma best practices and regulations of the future

Single-Arm Trials Using Real-World Evidence for Rare Disease Product Development

Logistics in Focus: Orchestrating the Challenges of Cell Therapy

World Orphan Drug Congresses Europe vs. The US: Global Perspectives on Genetic Testing

Understanding the Regulatory Environment in Japan for Generic Drug Development

Radiopharmaceutical deals drive sector growth despite supply crisis

BioMarin reports cancer case in hemophilia gene therapy trial

MEI and Kyowa Kirin to discontinue development of zandelisib outside Japan

Cannabinoids receptors: popular preclinical target but banned in 137 countries

Move to relax clinical trial norms will cut Indian pharma’s human studies cost, lower drug prices for patients

Study finds the metabolic regulator MYC is defective in obesity

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