ACTO

DECEMBER 19, 2023

The payoff of, on average, 15+ years of R&D and $2 billion invested to bring a new therapy to market comes down to what is done in the 180 days following a product launch. There is no margin for error, but there is no blueprint for success.

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The Pharma Data

APRIL 15, 2022

New GSK reaffirms its full-year 2022 guidance, the medium-term outlook for 2021-2026 of more than 5% sales and 10% adjusted operating profit CAGR* at CER**, and long-term sales ambition. billion (£1.5 Myelofibrosis is a fatal cancer of the bone marrow impacting the normal production of blood cells.

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The Pharma Data

NOVEMBER 11, 2020

and EU5 by 2026. The DSR therapy is still in development and it should be noted that any statements regarding safety and efficacy arise from ongoing pre-clinical and clinical investigations which have yet to be completed. The DSR therapy is not currently approved for clinical research in the United States or Canada.

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The Pharma Data

MAY 2, 2023

Starting in 2026, this facility will develop manufacturing processes for active pharmaceutical ingredients and produce drugs for use in clinical trial phases. This step is crucial for Boehringer Ingelheim to rapidly advance new drug candidates from the company’s vast research pipeline to clinical trials and ultimately to market approval.

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Small Molecule Clinical Trials Pharmaceuticals Pharmaceutical Companies 40

National Institute on Drug Abuse: Nora's Blog

SEPTEMBER 27, 2022

But while treatments such as medications for opioid use disorder (MOUD) or behavioral approaches like cognitive behavioral therapy or contingency management address the biological and behavioral dimensions of an SUD, changes in the brain and behavior take time, as does resolving the radiating impacts of an SUD on an individual’s life.

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The Pharma Data

DECEMBER 16, 2020

and EU5 by 2026. The DSR therapy is still in development and it should be noted that any statements regarding safety and efficacy arise from ongoing pre-clinical and clinical investigations which have yet to be completed. The DSR therapy is not currently approved for clinical research in the United States or Canada.

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The Pharma Data

OCTOBER 14, 2020

Pre-clinical and clinical proof-of-concept data from a first-in-human single dose DSR study demonstrated that single dose DSR therapy was safe and well-tolerated and the positive results were published in the high impact peer-reviewed cardiovascular journal, Circulation. and EU5 by 2026.

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The Pharma Data

NOVEMBER 18, 2020

The company continues to believe that, if positive, these trial data could represent a significant value inflection point for the company and its shareholders, confirming Betalutin® as a highly promising new targeted therapy that can address the unmet needs of R/R FL patients. Presentation and live webcast – Q3 2020 results.

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Agency IQ

AUGUST 16, 2024

However, the agency can describe a concomitant therapy in labeling for a product it has authority to regulate if it is essential for the therapeutic effect. While the ability to describe and specify aspects of the therapy is limited, the agency can specify the number and licensure of therapists who would participate in the MDMA treatment.

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The Pharma Data

DECEMBER 12, 2020

It plays a crucial role in many inflammatory and autoimmune diseases across multiple therapy areas, including haematology, nephrology, neurology, metabolic disorders, cardiology, ophthalmology and acute care. Rare diseases is a high-growth therapy area with rapid innovation and significant unmet medical need. AstraZeneca PLC. _.

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Agency IQ

AUGUST 2, 2024

While this would not affect existing vouchers, it would prevent FDA from issuing new ones after September 2026. 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.,

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The Pharma Data

NOVEMBER 11, 2020

Strategic alliance with Novo Nordisk to develop novel therapies for kidney diseases. Renewal of contract with CEO Dr Werner Lanthaler for five years until 05 March 2026 (after period-end). Renewal of contract with CEO Dr Werner Lanthaler for five years until 05 March 2026 (after period-end).

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Agency IQ

JUNE 9, 2023

High-priced cell and gene therapies are being increasingly developed for rare diseases. For example, a comparison of some gene therapy reimbursement shows that the single-treatment CAR-T cell therapy Kymriah (tisagenlecleucel) is reimbursed at 320,000 euros (based on outcome) in Italy, Germany, Spain and France.

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Marketing Regulations Biosimilars Drugs 40

Agency IQ

FEBRUARY 15, 2024

We have tried to sort guidance documents by topic area.

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Agency IQ

MAY 17, 2024

At that time, the FDA would no longer be able to award new vouchers to companies, unless the product already had rare pediatric disease designation, and obtained approval before October 1, 2026. Combination products could include standards of care or other therapies owned by the same sponsor as the primary drug.

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FDA Disease FDA Approval Production 40

National Institute on Drug Abuse: Nora's Blog

NOVEMBER 23, 2022

But even with multiple forms of HIV prevention now available, including pre-exposure prophylaxis (PrEP) pills for people who are HIV-negative and antiretroviral therapy that can help people with HIV maintain an undetectable viral load and thus not transmit the virus, HIV transmission rates remain frustratingly elevated.

Treatment 52

Treatment Virus Research Drugs 52

The Pharma Data

JANUARY 6, 2021

“We are leveraging our strengths in process development and manufacturing for a wide range of biopharmaceuticals such as antibodies, recombinant proteins, gene therapies and vaccines,” said Martin Meeson , chief executive officer at FUJIFILM Diosynth Biotechnologies.

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Codon

SEPTEMBER 29, 2024

It suggested that, in the future, certain classes of drugs, specifically genetic therapies, could be screened entirely virtually via models of life. Models trained on nucleotide data were ideal for understanding how genetic therapies altered cellular dynamics, so they powered the genetic editing revolution.

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The Pharma Data

SEPTEMBER 15, 2020

27 key programs highlighted, including assets that could potentially contribute revenue by 2025 and others in the 2026-2028 time frame. Based on the acceptable safety profile and the favorable immune response data, including the 4th dose response data, Pfizer received Breakthrough Therapy Designation.

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Drug Hunter

APRIL 24, 2023

A landmark part of the act is the requirement of Medicare to negotiate the prices for the 15 drugs with highest Medicare spend without generic and/or biosimilar substitutions across Part B (hospital drugs) and D (at-home drugs), with negotiated price implementation starting in 2026.

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The Pharma Data

JUNE 2, 2022

In July 2021, the US Food and Drug Administration granted Breakthrough Therapy designation for AFX3772 to prevent S. New GSK reaffirms its full-year 2022 guidance and the medium-term outlook for 2021-2026 of more than 5% sales and 10% adjusted operating profit CAGR* at CER**. Phase III is expected to start in the short term.

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Vaccine Immune Response Clinical Trials Disease 52

The Pharma Data

MAY 31, 2022

In July 2021, the US Food and Drug Administration granted Breakthrough Therapy designation for AFX3772 to prevent S. New GSK reaffirms its full-year 2022 guidance and the medium-term outlook for 2021-2026 of more than 5% sales and 10% adjusted operating profit CAGR* at CER**. Phase III is expected to start in the short term.

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Vaccine Immune Response Clinical Trials Disease 52

Drug Target Review

MARCH 20, 2025

The landscape of anticoagulation therapy could be on the brink of a major transformation. GI bleeding can lead to patients temporarily or permanently discontinuing their anticoagulation therapy, significantly increasing their stroke risk. “When someone has a GI bleed, they often stop taking their anticoagulant.

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Therapies Clinical Development Trials FDA 52

FDA Law Blog: Drug Discovery

FEBRUARY 11, 2025

On February 5, 2025, a press release caught our eye Arbor Biotechnologies announced that ABO-101, its investigational therapy for the treatment of primary hyperoxaluria type 1 (PH1), received a rare pediatric disease designation.

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Disease FDA Therapies Drugs 59

New Drug Approvals

APRIL 27, 2025

New Drug Therapy Approvals 2024 (PDF). Flurpiridaz F 18 WeightAverage: 367.84 3] Flurpiridaz ( 18 F) It is given by intravenous injection. [3] 3] Flurpiridaz ( 18 F) was approved for medical use in the United States in September 2024. [3] 2008 ; Nagel 2014 ) Briefly, to a mixture of mucochloric acid ( 1 ) (1.18 mmol) and Na 2 CO 3 (0.33

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FDA Clinical Trials FDA Approval Disease 57

FDA Law Blog: Biosimilars

FEBRUARY 4, 2025

This time, however, the program, which has helped incentivize the development of dozens of pediatric rare disease therapies since 2012, was not renewed. The authors suggested that the developers of such therapies would receive a priority review voucher that they can sell to manufacturers of blockbuster drugs. See Table 1.

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