Food and Drug Administration Safety and Innovation Act

FDA Grants Orphan Drug Designation to Amplyx Pharmaceuticals for APX001 for Treatment of Cryptococcosis

Retrieved on:

Monday, February 11, 2019

Health, Articles, Food and Drug Administration, Pharmaceuticals policy, Pharmacy, Pharmaceutical industry, Orphan drug, New Drug Application, Lipoglycopeptide, Food and Drug Administration Safety and Innovation Act

SAN DIEGO, Feb. 11, 2019 /PRNewswire/ --Amplyx Pharmaceuticals, a company developing first-in-class products for life-threatening infections, announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Product Development has granted orphan drug designation to APX001, the company's lead drug candidate, for treatment of cryptococcosis.

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SAN DIEGO, Feb. 11, 2019 /PRNewswire/ --Amplyx Pharmaceuticals, a company developing first-in-class products for life-threatening infections, announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Product Development has granted orphan drug designation to APX001, the company's lead drug candidate, for treatment of cryptococcosis.
Orphan drug designation qualifies APX001 for seven years of market exclusivity in the U.S. upon FDA approval of a new drug application (NDA) for the orphan designated indications.
In addition to orphan designations, FDA had previously granted Qualified Infectious Disease Product (QIDP) designation for APX001 for treatment of cryptococcosis, invasive candidiasis, invasive aspergillosis, and coccidioidomycosis.
Under the FDA's Orphan Drug Designation program, orphan drug designation is granted by the FDA to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the U.S.

US FDA Accepts Regulatory Submissions for Review of Tafamidis to Treat Transthyretin Amyloid Cardiomyopathy

Retrieved on:

Monday, January 14, 2019

Health, Biotechnology, Pharmaceutical, FDA, General Health, Health, Food and Drug Administration, Pharmaceuticals policy, Priority review, Orphan drugs, Prescription Drug User Fee Act, Pharmacy, Fast track, Tafamidis, Prescription drug, Synageva, Food and Drug Administration Safety and Innovation Act

The FDA grants Priority Review to medicines that may offer significant advances in treatment or may provide a treatment where no adequate therapy exists.

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The FDA grants Priority Review to medicines that may offer significant advances in treatment or may provide a treatment where no adequate therapy exists.
The target Prescription Drug User Fee Act (PDUFA) action date for a decision by the FDA is in July 2019.
We look forward to working with the FDA to bring the first treatment for this deadly disease to patients.
In June 2017 and May 2018, respectively, the FDA granted tafamidis Fast Track and Breakthrough Therapy designations for ATTR-CM.

Stimulated Development of Orphan Drug Designations for Rare Diseases and Anti-Cancer Drug Candidates Intensifies

Retrieved on:

Thursday, December 6, 2018

Health, Pharmaceuticals policy, Biotechnology, Food and Drug Administration, Drug discovery, Orphan drug, Orphan Drug Act, Food and Drug Administration Safety and Innovation Act

Treatments for rare diseases are often given a designation by the FDA known as "orphan".

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Treatments for rare diseases are often given a designation by the FDA known as "orphan".
There were 27 different therapeutic areas overall, with seven of those areas having 10 or more approved orphan drugs.
Anderson Cancer Center, today announced it has filed a request with the U.S. Food and Drug Administration ("FDA") for Orphan Drug Status for its drug candidate WP1066.
"Given its potential to address rare and difficult to treat cancers, including glioblastoma, we believe WP1066 is well positioned to qualify for Orphan Drug Status."

Aronora's New Blood Clot Treatment Receives FDA Fast Track Designation

Retrieved on:

Wednesday, September 26, 2018

Food and Drug Administration, Health, Fast track, Accelerated approval, Thrombus, Breakthrough therapy, Food and Drug Administration Safety and Innovation Act

However, tPA can significantly increase the risk of bleeding, so it is used only in a very small percentage of blood clot victims.

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However, tPA can significantly increase the risk of bleeding, so it is used only in a very small percentage of blood clot victims.
It's time for a new generation of safer emergency blood clot treatments, and we look forward to working closely with the FDA to develop this truly revolutionary drug candidate."
Fast Track designation is intended to support and expedite the development and approval of drugs that show promise in treating a serious or life-threatening disease.
Drugs with Fast Track designation can qualify for accelerated approval and frequent communication with the FDA throughout the development process.

Statement from FDA Commissioner Scott Gottlieb, M.D., on agency's continued efforts relating to compounded drugs for patients who cannot use an FDA-approved drug

Retrieved on:

Thursday, June 28, 2018

Health, Food and Drug Administration, Compounding, Pharmacy, Pharmaceuticals policy, Drug Safety, Bioidentical hormone replacement therapy, Food and Drug Administration Safety and Innovation Act

But because compounded drugs are not FDA-approved, it's important for patients and the medical community to have access to information about the quality of these products.

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But because compounded drugs are not FDA-approved, it's important for patients and the medical community to have access to information about the quality of these products.
In achieving all of these goals, we're committed to striking the balance Congress intended between access and safety.
That means taking actions to protect patients and enforce the existing laws that govern compounding.
In addition, the FDA is inspecting compounding facilities to assess whether drugs that are essentially copies of FDA-approved drugs are being compounded for patients who could use an FDA-approved drug.