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Despite recent advances in gene therapy for sickle cell disease (SCD) , automated red blood cell exchange (aRBCX) remains a cornerstone therapy that plays a vital yet underutilised role in managing complications and enhancing quality of life for millions living with this devastating condition worldwide.
Tobolowsky — The Rare Pediatric Disease Priority Review Voucher program has had a bit of a tumultuous history in its 12 short years of existence. Over 50 rare pediatric disease priority review vouchers have been awarded to date. Notably, it was reauthorized in 2016 (until 2020) and in 2020 (until 2024).
2] Fitusiran was approved for medical use in the United States in March 2025. [2] 1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] 26 March 2025.
5] Leniolisib was approved for medical use in the United States in March 2023. [5] 5] [7] [8] It is the first approved medication for the treatment of activated PI3K delta syndrome. [5] 5] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. [9] World Health Organization (2016).
based subsidiary of Terumo and a global neurovascular company, announced today the FDAApproval of the PMA Supplement for the WEB 17 System, a new addition to the WEB Aneurysm Embolization System for the treatment of intracranial wide neck bifurcation aneurysms. .
ALISO VIEJO, Calif. ,
A rare, or orphan, disease by definition affects a small percentage of the population — fewer than 200,000 people in the U.S. But the numbers add up, and taken together, rare diseases impact an estimated 30 million Americans. Food and Drug Administration (FDA) approval. The FDA Since 1983. FDA Expedited Programs.
Metabolism of 2023 FDAApproved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. link] [3] FDA prescribing information for daprodustat. Basic Clin Pharmacol Toxicol. 131(5): 311- 324.
Food and Drug Administration (FDA) has now approved the very first oral drug to treat the millions of men affected. . Myovant was launched in 2016 as one of Vivek Ramaswamy’s family of biotech Vants after a deal with Takeda for a prostate cancer drug and a female infertility drug. compared to AbbVie’s at 6.2%.
A healthy immune system defends the body against disease and other conditions. Autoimmune disease impacts different parts of the body, weakening functionality. Researchers are aware of more than 80 diseases that occur when the immune system attacks the body’s own organs, tissues and cells. It may be life-threatening.
HSV in immunocompromised patients Although HSV infection is very common in the general population, it rarely causes serious disease and is effectively contained by the immune system. 7] If the virus also acquires resistance to foscarnet, then there is currently no FDAapproved treatment. References ^ Wilck, M.B.; PMID 23465005.
People with type 2 diabetes are at increased risk for heart attacks, stroke, and other forms of cardiovascular disease, and at an earlier age than other people. In fact, the evidence suggests that such drugs might even offer some protection against heart disease. 2016 Jun 1;8(341):341ra76. [3] December 2008. [2] Sci Transl Med.
District Court challenging FDA’s plan to remove their products from the market. Specifically, FDA is looking to remove a drug called carbadox from the market. Used for over 50 years, carbadox is an antimicrobial drug used to treat gastrointestinal disease in pigs. 360b(d)(1)(I).
Explain the FDAApproval Process Many patients are unaware of the rigorous approval process generic drugs must undergo. Educate them about the FDA’s role in ensuring the safety and efficacy of generic medications. . ” JAMA, 2016. World Health Organization. ” WHO.int, 2020. Shrank, W.H.,
v=uT2-0qoHjbc Who will be there: Molecule & VitaDAO Core Teams If you haven’t yet read up on the research project as well as the researchers behind it, find more about it below: About the Research Project What if therapeutics to slow down the ageing process and prevent age-related disease already existed? billion prescriptions from 4.8
Credit: Brittany Mahoney As NIH Director, I often hear stories of how people with serious diseases—from arthritis to Zika infection—are benefitting from the transformational power of NIH’s investments in basic science. Credit: Zhang & Chen, 2016, Cell 167, 1586–1597. First, a bit of history! About 30,000 Americans have CF.
MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs. What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies?
If approved, Xarelto will be the only oral Factor Xa Inhibitor indicated in the U.S. There are currently no FDA-approved anticoagulation therapies for pediatric patients with congenital heart disease who have undergone the Fontan procedure. for use in pediatric patients. EINSTEIN-Jr. About the EINSTEIN-Jr.
The approach might also be put to use for optimizing drug candidates or for discovering new uses for cancer drugs that have already been FDAapproved. 2016 Feb 29. With PRISM, it’s now feasible to screen entire small molecule libraries across large panels of cancer cell lines. Nat Biotechnol.
In 2016, the Food and Drug Administration (FDA) approved Spinraza (nusinersen). It was the first drug to treat children with spinal muscular atrophy (SMA), a disease affecting children’s muscular strength and movement. While the FDA’sapproval of nusinersen may not seem extraordinary, it was.
Dupixent plus TCS reduced overall disease severity by 70% and itch by 49%. Results reinforce well-established safety profile of Dupixent – the first ever biologic medicine for atopic dermatitis currently approved for patients as young 6 years old. Yancopoulos, M.D., President and Chief Scientific Officer at Regeneron. “
The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDAapproval. If the confirmatory trial shows that the drug actually provides a clinical benefit, then the FDA grants traditional approval for the drug. 8,128,929 was submitted on December 8, 2016 (Docket No.
Based on a technology developed by Broad Institute core member David Liu’s laboratory, the treatment is the first in a series of new medicines being tested to treat rare diseases by repairing patients’ particular genetic misspellings. s to be produced at scale, making them the standard of care for life-threatening rare genetic diseases.
BOTOX ® Cosmetic is the first and only product of its kind FDA-approved to treat three areas, temporarily improving the appearance of moderate to severe forehead lines, glabellar lines and crow’s feet in adults.
Data on File, Allergan, 2016; Clinical Study Report Satisfaction Table.
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Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.
Until recently, the FDA relied on a monograph process through which firms could bring OTC drugs to market without FDAapproval so long as it adhered to pre-set terms under the monograph. Four comments were submitted to the docket established for the 2015 Petition.
The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDAapproval, in 2023. Gray, in London to discuss the significance of her recovery at the Third International Summit on Human Genome Editing , described Casgevy as “a new beginning for people with sickle cell disease.”
FDA conducted the eight-factor scheduling analysis required by the CSA in 2016 and found that marijuana continued to meet the scheduling criteria for remaining in schedule I. 12, 2016); Denial of Petition to Initiate Proceedings to Reschedule Marijuana, 81 Fed. 53,688 (Aug. 53,767 (Aug. 21 U.S.C. § at 53,700; 81 Fed.
First approval of a conjugate vaccine that helps protect against 20 serotypes responsible for the majority of invasive pneumococcal disease and pneumonia, 1,2,3,4,5,6,7 including seven responsible for 40% of pneumococcal disease cases and deaths in the U.S. Following today’s FDAapproval, the U.S. Jansen, Ph.D.,
He started to imagine how the deadly and contagious disease, if confirmed, might spread to half the city’s population. Eight of the 20 patients died, but the spread of the disease in Nigeria stopped there. Blood and urine samples from the man were waiting for Happi in his lab. Happi felt chilled. They called the idea Sentinel.
FDA/HHS Analysis, 2023 FDA/HHS last conducted an eight-factor analysis of marijuana in 2016 and concluded that marijuana continued to meet schedule I criteria. 12, 2016); Denial of Petition to Initiate Proceedings to Reschedule Marijuana, 81 Fed. Denial of Petition to Initiate Proceedings to Reschedule Marijuana, 81 Fed.
In early August, risdiplam, an orally available small molecule that binds to a stem-loop structure in the SMN2 pre-mRNA, received FDAapproval for the treatment of spinal muscular atrophy , a devastating genetic disease. In less severe disease, patients will reach adulthood and suffer from motor function deficiencies.
In December, Gilead shifted from bolstering its oncology business and strengthened its liver disease pipeline with the $1.4 AstraZeneca Dives into Rare Diseases: AstraZeneca has been in the spotlight for the COVID-19 vaccine candidate it developed with Oxford University. But the U.K.-based
If approved, XARELTO ® will be the first and only oral Factor Xa inhibitor indicated in the U.S. There are currently no FDA-approved anticoagulation therapies for pediatric patients with congenital heart disease who have undergone the Fontan procedure, a surgical procedure that redirects blood flow from the lower body to the lungs.
Five other countries participated in a concurrent submission and review process through FDA’s Project Orbis. This approval reinforces how critical it is to test all lung cancer patients for EGFR mutations before deciding how to treat them and regardless of their stage at diagnosis. 0.23; p<0.0001). 0.23; p<0.0001).
The target action date for the FDA decision on this investigational use is June 9, 2022. Dupixent remains the only biologic medicine approved for patients 6 years of age and older in this indication. Safety results were generally consistent with the safety profile of Dupixent in atopic dermatitis for patients aged 6 years and older.
“The results add to the extensive clinical evidence supporting the use of single-agent ibrutinib for long-term disease control.” IMBRUVICA ® is now approved in 101 countries and has been used to treat more than 230,000 patients worldwide across its approved indications. Barr , M.D., 1 Additionally, at 6.5
12/29/2023 FDORA, Section 3607 Clinical Trials Modernization : FDA must issue or revise draft guidance regarding “the use of seamless, concurrent, and other innovative clinical trial designs.” Within 180 days, FDA will release a report on the proceedings of the public meeting and any recommendations.
An update on the Medical Device Quality Management System Regulation : In February 2022, the FDA proposed a new rule which would effectively transition the agency away from its longstanding Quality System Regulation (QSR) in favor of the ISO 13485:2016 standard. Read our analysis of that rule here and here. ]
Dr. Karl Dussik, an Austrian physician, was among the first to suggest that ultrasound could be used to diagnose diseases within the body in 1941. By 2016, Shapiro had set up a research laboratory at the California Institute of Technology in Pasadena and began to devote his attention entirely to gas vesicles.
In 2012, an Israeli company called Protalix gained approval for their plant-based enzyme product for Gaucher’s disease. In 2016, a company called Synageva BioPharma (formerly AviGenics) was acquired by Alexion for $8.4B. ” Finally, “pharmed” drugs are making their way onto the market.
We wondered how given that HHS and the Drug Enforcement Administration (“DEA”) conducted eight-factor scheduling analyses in 2016, concluding that there was “no substantial evidence that marijuana should be removed from Schedule I.” 12, 2016) ; Denial of Petition to Initiate Proceedings to reschedule Marijuana, 81 Fed. 12, 2016).
New indication allows this first FDA-approved biomarker-based test to be used as triage for positive cobas HPV tests run on cobas 6800/8800 Systems in primary screening or co-testing programs. in 2020, according to the American Cancer Society, and about 4,290 women will die from the disease this year.
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