2016, Disease and FDA Approval - Drug Discovery Digest

2016

Disease

FDA Approval

Automated red blood cell exchange: bridging treatment gaps in sickle cell disease care

Drug Target Review

JUNE 5, 2025

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.

Disease

Disease Treatment Therapies Nurses

Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

Broad Institute

JUNE 24, 2025

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.

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Fitusiran

New Drug Approvals

APRIL 11, 2025

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.

Clinical Trials

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Leniolisib

New Drug Approvals

MAY 15, 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).

FDA Approval

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A Visual Guide to Genome Editors

Codon

JUNE 5, 2025

The treatment, now known as Casgevy, became the first CRISPR-based therapy to gain FDA approval, 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.”

DNA

DNA RNA Clinical Trials Engineering

The Long Road to End Tuberculosis

Codon

NOVEMBER 3, 2024

Physicians working in the early 20th century had little choice but to treat the world’s most rampant infectious disease with methods such as these. But even now, more than a century later, TB remains the deadliest infectious disease on Earth, killing about 1.2 million people every year. coli bacteria from growing.

Vaccine

Vaccine Trials Therapies Treatment

Ropotrectinib

New Drug Approvals

AUGUST 7, 2025

was granted FDA approval on November 15, 2023. Repotrectinib, a novel tyrosine kinase inhibitor (TKI), is the pioneering drug developed to specifically target ROS1 or NTRK-positive metastatic NSCLC and effectively combat the primary factors contributing to disease advancement [90].Preparation 76 (2016) 2132. [88]

FDA

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PRITELIVIR MESYLATE

New Drug Approvals

JULY 8, 2025

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 FDA approved treatment. References ^ Wilck, M.B.; PMID 23465005.

Virus

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A Temporary Extension for the Rare Pediatric Disease Priority Review Voucher with a Longer-Term Extension in Sight?

FDA Law Blog: Biosimilars

OCTOBER 13, 2024

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).

Disease

Disease FDA Approval FDA Government

Educating Patients about Generic Drugs: Strategies for Success

Drug Patent Watch

JANUARY 7, 2025

Explain the FDA Approval 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.,

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Lower-Profile Web Intrasaccular Aneurysm Treatment Device Receives FDA Approval

The Pharma Data

JANUARY 11, 2021

based subsidiary of Terumo and a global neurovascular company, announced today the FDA Approval 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. ,

FDA Approval

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An Evolving Regulatory Environment for Rare and Orphan Diseases

Advarra

AUGUST 16, 2022

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.

Disease

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Metabolism of 2023 FDA Approved Small Molecules – PART 1

Metabolite Tales Blog

JANUARY 23, 2024

Metabolism of 2023 FDA Approved 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.

Small Molecule

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FDA Approves Myovant Drug as First-Ever Oral Hormone Therapy for Prostate Cancer

The Pharma Data

DECEMBER 21, 2020

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%.

FDA Approval

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Advances in the Battle Against Autoimmune Disease

The Pharma Data

DECEMBER 15, 2020

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.

Disease

Disease Clinical Trials Small Molecule Trials

Precision Medicine: Using Genomic Data to Predict Drug Side Effects and Benefits

NIH Director's Blog: Drug Development

JUNE 14, 2016

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.

Drugs

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From the Porcine to the Ridiculous: Veterinary Drugmaker Sues FDA for Alleged APA Violations

FDA Law Blog: Biosimilars

JANUARY 18, 2024

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).

FDA

FDA FDA Approval International Production

Announcing the first biopharma IPNFT Transaction

Molecule Blog

AUGUST 13, 2021

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

FDA Approval

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Another Milestone in the Cystic Fibrosis Journey

NIH Director's Blog: Drug Development

JULY 20, 2017

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.

Clinical Trials

Clinical Trials Disease Small Molecule Therapies

Advancing CAR-T therapy: how CD5 modulation is shaping cancer treatment

Drug Target Review

AUGUST 29, 2024

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?

Therapies

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FDA for approval to treat VTE and to prevent VTE in children

The Pharma Data

JUNE 28, 2021

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.

FDA

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Clover Biopharmaceuticals Announces Publication of Phase 1 Clinical Trial Data for its Adjuvanted COVID-19 Vaccine Candidates in The Lancet

The Pharma Data

JANUARY 31, 2021

Many major disease targets are trimerization-dependent such as the tumor necrosis factor (TNF) superfamily (involved in extrinsic apoptosis, immune co-stimulation and inflammation) as well as enveloped RNA virus antigens responsible for entry into host cells. . CHENGDU, China , Feb. About Trimer-Tag© Technology.

Clinical Trials

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DNA Barcodes Could Streamline Search for New Drugs to Combat Cancer

NIH Director's Blog: Drug Discovery

MARCH 8, 2016

The approach might also be put to use for optimizing drug candidates or for discovering new uses for cancer drugs that have already been FDA approved. 2016 Feb 29. With PRISM, it’s now feasible to screen entire small molecule libraries across large panels of cancer cell lines. Nat Biotechnol.

DNA

DNA Drugs Small Molecule FDA Approval

Understanding FDA’s Draft Guidance on Ethical Considerations for Involving Children in Clinical Trials

Advarra

NOVEMBER 17, 2022

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’s approval of nusinersen may not seem extraordinary, it was.

Clinical Trials

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Dupixent® (dupilumab) pivotal trial meets all primary and secondary endpoints.

The Pharma Data

AUGUST 30, 2021

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. “

Trials

Trials Disease Treatment Clinical Trials

Clover Biopharmaceuticals Announces Positive Phase 1 Data for its Adjuvanted S-Trimer COVID-19 Vaccine Candidates

The Pharma Data

DECEMBER 3, 2020

Many major disease targets are trimerization-dependent such as the tumor necrosis factor (TNF) superfamily (involved in extrinsic apoptosis, immune co-stimulation and inflammation) as well as enveloped RNA virus antigens responsible for entry into host cells. About Trimer-Tag © Technology. View source version on businesswire.com: [link].

Vaccine

Vaccine Immune Response Virus RNA

Surely You Must be Kidding, PTO?!? “No, and Don’t Call Me Shirley!” – The Seemingly Slapstick (But Yet Unfunny) World of Recent Patent Term Extension Decisions (PART 2)

FDA Law Blog: Biosimilars

FEBRUARY 29, 2024

The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval. 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.

FDA

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Allergan Aesthetics and BOTOX® Cosmetic (onabotulinumtoxinA) Put Real Stories at the Forefront in New Campaign

The Pharma Data

JUNE 18, 2021

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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The genetic modifier approach: identifying the right target for rare diseases

Drug Target Review

JUNE 21, 2023

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.

Disease

Disease Clinical Trials Therapies Science

Analysis Life Sciences Thank You FDA’s nonprescription advisors find no efficacy for phenylephrine

Agency IQ

SEPTEMBER 15, 2023

Until recently, the FDA relied on a monograph process through which firms could bring OTC drugs to market without FDA approval so long as it adhered to pre-set terms under the monograph. Four comments were submitted to the docket established for the 2015 Petition.

Science

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Joint Basis for FDA/HHS Marijuana Rescheduling Recommendation Unveiled

FDA Law Blog: Biosimilars

JANUARY 18, 2024

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.

FDA

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U.S. FDA Approves PREVNAR 20™, Pfizer’s Pneumococcal 20-valent Conjugate Vaccine for Adults Ages 18 Years or Older

The Pharma Data

JUNE 18, 2021

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 FDA approval, the U.S. Jansen, Ph.D.,

Vaccine

Vaccine FDA Approval FDA Disease

A new viral surveillance system in West Africa is showing the world how to prevent the next pandemic

Broad Institute

APRIL 25, 2024

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.

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Virus Hospitals Disease Government

DEA Concurs: Marijuana Meets Schedule III Criteria

FDA Law Blog: Biosimilars

MAY 5, 2024

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.

FDA

FDA Regulations Treatment Licensing

A new drug approval for the vanguard of RNA-targeted small molecules

Dark Matter Blog

AUGUST 27, 2020

In early August, risdiplam, an orally available small molecule that binds to a stem-loop structure in the SMN2 pre-mRNA, received FDA approval 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.

Small Molecule

Small Molecule RNA Clinical Trials Drugs

Top 2020 Biopharma M&A Deals

The Pharma Data

DECEMBER 30, 2020

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

Science

Science Disease Pharmaceuticals Treatment

XARELTO® (rivaroxaban) to Help Prevent and Treat Blood Clots in Pediatric Patients

The Pharma Data

JUNE 26, 2021

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.

Doctors

Doctors Hospitals Treatment Clinical Trials

Tagrisso Approved in the US for the Adjuvant Treatment of Patients with Early-Stage EGFR-Mutated Non-Small Cell Lung Cancer

The Pharma Data

DECEMBER 21, 2020

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).

Treatment

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FDA accepts Dupixent® (dupilumab) for Priority Review in children aged 6 months to 5 years with moderate-to-severe atopic dermatitis

The Pharma Data

FEBRUARY 13, 2022

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.

FDA

FDA Disease Treatment FDA Approval

Results from IMBRUVICA® (ibrutinib) RESONATE-2 Study Provide Up to Seven Years of Progression-Free and Overall Survival Data in First-Line Chronic Lymphocytic Leukemia (CLL)

The Pharma Data

JUNE 18, 2021

“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

FDA Approval

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Analysis Life Sciences Thank You What We Expect the FDA to do in November 2023

Agency IQ

OCTOBER 27, 2023

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.

FDA

FDA Science Regulations Production

Analysis Life Sciences Thank You The 51 regulations that FDA is currently working on

Agency IQ

JUNE 16, 2023

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. ]

Regulations

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The Promise of Gas Vesicles

Codon

JUNE 18, 2024

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.

Engineering

Engineering Laboratories International Clinical Trials

Beyond Steel Tanks

Codon

MARCH 24, 2024

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.

Vaccine

Vaccine Engineering Production RNA

Automated red blood cell exchange: bridging treatment gaps in sickle cell disease care

Q&A: One scientist’s bold vision to make on-demand treatments routine for life-threatening rare genetic diseases

Trending Sources

Fitusiran

Leniolisib

A Visual Guide to Genome Editors

The Long Road to End Tuberculosis

Ropotrectinib

PRITELIVIR MESYLATE

A Temporary Extension for the Rare Pediatric Disease Priority Review Voucher with a Longer-Term Extension in Sight?

Educating Patients about Generic Drugs: Strategies for Success

Lower-Profile Web Intrasaccular Aneurysm Treatment Device Receives FDA Approval

An Evolving Regulatory Environment for Rare and Orphan Diseases

Metabolism of 2023 FDA Approved Small Molecules – PART 1

FDA Approves Myovant Drug as First-Ever Oral Hormone Therapy for Prostate Cancer

Advances in the Battle Against Autoimmune Disease

Precision Medicine: Using Genomic Data to Predict Drug Side Effects and Benefits

From the Porcine to the Ridiculous: Veterinary Drugmaker Sues FDA for Alleged APA Violations

Announcing the first biopharma IPNFT Transaction

Another Milestone in the Cystic Fibrosis Journey

Advancing CAR-T therapy: how CD5 modulation is shaping cancer treatment

FDA for approval to treat VTE and to prevent VTE in children

Clover Biopharmaceuticals Announces Publication of Phase 1 Clinical Trial Data for its Adjuvanted COVID-19 Vaccine Candidates in The Lancet

DNA Barcodes Could Streamline Search for New Drugs to Combat Cancer

Understanding FDA’s Draft Guidance on Ethical Considerations for Involving Children in Clinical Trials

Dupixent® (dupilumab) pivotal trial meets all primary and secondary endpoints.

Clover Biopharmaceuticals Announces Positive Phase 1 Data for its Adjuvanted S-Trimer COVID-19 Vaccine Candidates

Surely You Must be Kidding, PTO?!? “No, and Don’t Call Me Shirley!” – The Seemingly Slapstick (But Yet Unfunny) World of Recent Patent Term Extension Decisions (PART 2)

Allergan Aesthetics and BOTOX® Cosmetic (onabotulinumtoxinA) Put Real Stories at the Forefront in New Campaign

The genetic modifier approach: identifying the right target for rare diseases

Analysis Life Sciences Thank You FDA’s nonprescription advisors find no efficacy for phenylephrine

Joint Basis for FDA/HHS Marijuana Rescheduling Recommendation Unveiled

U.S. FDA Approves PREVNAR 20™, Pfizer’s Pneumococcal 20-valent Conjugate Vaccine for Adults Ages 18 Years or Older

A new viral surveillance system in West Africa is showing the world how to prevent the next pandemic

DEA Concurs: Marijuana Meets Schedule III Criteria

A new drug approval for the vanguard of RNA-targeted small molecules

Top 2020 Biopharma M&A Deals

XARELTO® (rivaroxaban) to Help Prevent and Treat Blood Clots in Pediatric Patients

Tagrisso Approved in the US for the Adjuvant Treatment of Patients with Early-Stage EGFR-Mutated Non-Small Cell Lung Cancer

FDA accepts Dupixent® (dupilumab) for Priority Review in children aged 6 months to 5 years with moderate-to-severe atopic dermatitis

Results from IMBRUVICA® (ibrutinib) RESONATE-2 Study Provide Up to Seven Years of Progression-Free and Overall Survival Data in First-Line Chronic Lymphocytic Leukemia (CLL)

Analysis Life Sciences Thank You What We Expect the FDA to do in November 2023

Analysis Life Sciences Thank You The 51 regulations that FDA is currently working on

The Promise of Gas Vesicles

Beyond Steel Tanks

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