Rare Disease Drug Approvals

Listing Websites about Rare Disease Drug Approvals

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Rare Disease and Orphan Drug Designated Approvals FDA

(7 days ago) CY 2015 CDER Rare Disease and Orphan Drug Designated Approvals. CY 2014 CDER Rare Disease and Orphan Drug Designated Approvals. CY 2013 CDER Rare Disease and …

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Category:  Health Detail Drugs


CDER Continues to Make Rare Diseases a Priority with …

(4 days ago) Over the past decade or so, we have seen an upward trajectory in the percentage of drugs approved to treat rare or “orphan” diseases. Last year, 26 of CDER’s 50 novel drug …

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Category:  Health Detail Drugs


Rare Diseases at FDA FDA - U.S. Food and Drug …

(6 days ago) Since the Orphan Drug Act was signed into law in 1983, the FDA has approved hundreds of drugs for rare diseases, but most rare diseases do not have FDA-approved treatments. The …

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Category:  Health Detail Drugs


Insights into Rare Disease Drug Approval: Trends and …

(7 days ago) approvals to positively impact the rare diseases they treat. • Biologics, rare cancer treatments, and targeted therapies are reflected in a growing proportion of orphan drug …

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Category:  Cancer Detail Drugs


FDA highlights 2020 rare disease drug approvals - Med Journal 360

(5 days ago) In 2020, the CDER approved 53 novel drugs. More than half of those (31) were new approval for drugs to treat patients with rare diseases. A number of novel therapies for rare cancers, …

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Category:  Cancer Detail Drugs


Rare Disease Day 2021: FDA Shows Sustained Support of …

(9 days ago) Progress in Orphan Drug and Rare Pediatric Disease Designations. there is still much work to be done as the vast majority of rare diseases do not have approved treatments. In 2021, we …

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Category:  Health Detail Drugs


Rare Diseases and Disorders FDA Approved Drugs

(1 days ago) FDA Approved Drugs. The following database contains a listing of drugs approved by the Food and Drug Administration (FDA) for sale in the United States. Drug …

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Category:  Health Detail Drugs


Novel Drug Approvals for 2020 FDA

(4 days ago) 11/25/2020. To treat high-risk refractory or relapsed neuroblastoma. Drug Trials Snapshot. 45. Imcivree. setmelanotide. 11/25/2020. To treat obesity and the control of hunger associated …

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Category:  Health Detail Drugs


2022 Orphan Drugs: PDUFA Dates and FDA Approvals

(Just Now) Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and …

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Category:  Medications Detail Drugs


Ultra-rare disease specialists top 2021's pricey drugs list, …

(2 days ago) Eiger BioPharmaceuticals in November snagged FDA clearance for Zokinvy for ultra-rare rapid-aging diseases that affect an estimated 600 people worldwide. Orphan drugs that treat small patient

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Category:  Pharmaceutical Detail Drugs


Boehringer bags first approval for rare skin disease drug

(8 days ago) September 2, 2022. German drugmaker Boehringer Ingelheim has its first regulatory approval for spesolimab, its antibody therapy for rare and life-threatening skin …

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Category:  Health Detail Drugs


New Drug Therapy Approvals 2020 FDA

(Just Now) In 2020, 31 of CDER’s 53 novel drug approvals, (58%) were approved to treat rare or “orphan” diseases that affect 200,000 or fewer Americans. Patients with rare …

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Category:  Health Detail Drugs


The Rare Disease Market: Policy Changes and Coverage Trends

(8 days ago) With the growing number of drugs approved to treat rare diseases, payers are exploring various methods to address critical issues related to pricing, value, novel payment, …

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Category:  Health Detail Drugs


Use of real-world evidence in regulatory decisions for rare …

(1 days ago) Lastly, we discuss future directions to expand the use of RWD and RWE to support orphan drug approvals, including the need for including patient experience data as an important source of …

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Category:  Health Detail Drugs


New Study Investigates the Number of Available Orphan Products

(3 days ago) While most rare diseases do not yet have a n FDA-approved treatment, some have multiple treatments available due to the presence of generics and biosimilars. At the time …

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Category:  Health Detail Drugs


CDER Continues to Make Rare Diseases a Priority with Drug …

(7 days ago) The combination of government incentives and scientific advancements has fueled extraordinary development of drugs for rare diseases. CDER’s Approvals for Rare Diseases. Over the …

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Category:  Health Detail Drugs


FDA Approves A Drug For A Rare Disease That Causes Children To …

(5 days ago) A newly approved drug can extend the lives of children with progeria, a rare disorder that causes rapid aging. The drug is the result of one family's effort to help a child …

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Category:  Health Detail Drugs


FDA’S ACCELERATED APPROVAL PATHWAY: A RARE DISEASE …

(3 days ago) because of accelerated approvals. For rare disease patients specifically, accelerated approval offers a valuable source of hope. Over 25 million Americans suffer from rare diseases and, of …

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Category:  Health Detail Drugs


Cost of Amylyx ALS drug revives pricing debate for new meds in …

(9 days ago) The $158,000 a year wholesale price of Relyvrio falls just below the annual cost of Radicava, a Mitsubishi Tanabe Pharma ALS drug that was approved by the FDA in 2017. But …

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Category:  Health Detail Drugs


Sanofi, after long research journey, wins FDA approval for rare …

(5 days ago) Sanofi has won Food and Drug Administration approval to sell Xenpozyme, the first treatment specifically for an extremely rare genetic disease known as acid …

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Category:  Health Detail Drugs


Search Orphan Drug Designations and Approvals

(5 days ago) FDA Home. Developing Products for Rare Diseases & Conditions. This page searches the Orphan Drug Product designation database. Searches may be run by entering the product …

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Category:  Health Detail Drugs


FDA panel backs much-debated ALS drug in rare, 2nd review

(Just Now) The Food and Drug Administration advisers voted 7-2 that data from Amylyx Pharma warranted approval, despite ongoing concerns about the strength and reliability of the …

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Category:  Health Detail Drugs

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FAQ about Rare Disease Drug Approvals

Are there FDA-approved drugs for rare diseases?

Since the Orphan Drug Act was signed into law in 1983, the FDA has approved hundreds of drugs for rare diseases, but most rare diseases do not have FDA-approved treatments. The FDA works with many people and groups, such as patients, caregivers, and drug and device manufactures, to support rare disease product development.

Are there any orphan drugs approved by the FDA?

Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory dates for all orphan drugs for 2022.

Why are clinical trials difficult for patients with rare diseases?

The inherently small population of patients with a rare disease can also make conducting clinical trials difficult. Since the Orphan Drug Act was signed into law in 1983, the FDA has approved hundreds of drugs for rare diseases, but most rare diseases do not have FDA-approved treatments.

What are the most common approvals for neurological drugs?

Approvals of note for patients with rare neurological diseases include: First orally-administered drug to treat patients with spinal muscular atrophy, a disease that diminishes physical strength by affecting nerve cells in the spinal cord, taking away the ability to walk, eat, or breathe