SugarCone Biotech

JUNE 19, 2025

Typical treatment has been chemotherapy and radiotherapy with an initially high overall response rate (ORR) but then rapid recurrence followed by poor prognosis. Tarlatamab triggered an ORR of ~40% in refractory (second-line or later) SCLC patients in the DeLLphi-301 clinical trial published in 2023. months (versus 20% ORR and 8.3

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The Pharma Data

JUNE 25, 2025

FDA Approves Label Update for Lilly’s Amyvid, Expanding Its Role in Alzheimer’s Disease Diagnosis and Therapy Guidance In a major development for Alzheimer’s diagnostics, Eli Lilly and Company (NYSE: LLY) announced that the U.S.

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The Pharma Data

JUNE 9, 2025

Xywav: A Low-Sodium Alternative with FDA Approval Xywav is a uniquely formulated, low-sodium oxybate therapy, and remains the only product of its kind approved by the U.S. It is also approved for adult patients with idiopathic hypersomnia (IH). According to Dr. Richard J. Notably, the safety profile remained favorable.

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New Drug Approvals

MAY 17, 2025

Tofersen , sold under the brand name Qalsody , is a medication used for the treatment of amyotrophic lateral sclerosis (ALS). [3] Jump up to: a b c d e f g h i j k l “FDA approves treatment of amyotrophic lateral sclerosis associated with a mutation in the SOD1 gene” (Press release). 25 April 2023.

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New Drug Approvals

JULY 9, 2025

Sunvozertinib , sold under the brand name 舒沃哲, among others is an anti-cancer medication used for the treatment of non-small-cell lung cancer. [2] 2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1] 2] [4] Sunvozertinib was approved for medical use in the United States in July 2025. [1]

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The Pharma Data

JUNE 20, 2025

FDA Approves Dupixent as the First and Only Targeted Therapy for Adults with Bullous Pemphigoid In a groundbreaking development for patients suffering from a rare and debilitating autoimmune skin disease, the U.S. For many years, systemic corticosteroids have been the mainstay of BP treatment. receiving placebo.

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The Pharma Data

JUNE 25, 2025

Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to riliprubart , an investigational immunology therapy developed by Sanofi, for the treatment of antibody-mediated rejection (AMR) in solid organ transplantation.

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New Drug Approvals

APRIL 11, 2025

2] Fitusiran was approved for medical use in the United States in March 2025. [2] 2] The label also has a warning about liver toxicity and the need to monitor liver blood tests at baseline and then monthly for at least six months after initiating treatment with fitusiran or after a dose increase of fitusiran. [2] Fitusiran 1711.0g/mol,

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New Drug Approvals

JUNE 11, 2025

4] It was approved for the treatment of major depressive disorder in the United States in September 2023. [4] 4] This came after the drug had been rejected by the Food and Drug Administration (FDA) three times over two decades due to insufficient evidence of effectiveness. [5] 1] It is taken orally. [1] 1] It is taken orally. [1]

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Broad Institute

JULY 21, 2025

There is no cure or effective treatment for this rare genetic disease, but new research suggests a potential path to one.& The team used a scalable approach to develop prime editing treatments that directly repaired five different AHC-causing genetic mutations. The results really exceeded our expectations,” said Sakai. “It

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New Drug Approvals

APRIL 25, 2025

3] Inavolisib was approved for medical use in the United States in October 2024. [3] 3] Inavolisib was approved for medical use in the United States in October 2024. [3] 8] Therefore, it may serve as a new addition to combination therapy with conventional cancer treatment, such as chemotherapy. 3 November 2006.

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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. Some of these treatments, like K.J.’s, The team that treated K.J. Today, K.J. Today, K.J.

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New Drug Approvals

MAY 9, 2025

2] Elacestrant was approved for medical use in the United States in January 2023, [1] [2] [5] [6] and in the European Union in September 2023. [3] Randomization was stratified based on whether the ESR1 mutation was detected or not, prior treatment with fulvestrant, and presence of visceral metastasis. [2] 1] [4] It is taken by mouth.

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Broad Institute

MAY 21, 2025

The work of June, Levine, Rivire, and Sadelain laid the foundation for a whole new class of treatments. In the early 2000s, both teams began preparing for clinical trials. Their contributions have saved lives and opened doors for future innovations that will continue to transform human health.

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New Drug Approvals

APRIL 2, 2025

1] [2] It was developed by Vertex Pharmaceuticals , [5] and was approved for medical use in the United States in January 2025. [2] 2] [6] Suzetrigine is the first medication to be approved by the US Food and Drug Administration (FDA) in this new class of pain management medicines. [2] Suzetrigine is taken by mouth. [1]

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The Pharma Data

JUNE 15, 2025

According to the data, the combination demonstrated a high ORR and a strong depth of response in a patient population with limited treatment options and poor outcomes under standard care. were penta-drug refractory, having been exposed to additional lines of treatment. Among these: 84.4% had previously received a bispecific antibody.

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The Premier Consulting Blog

JANUARY 14, 2025

The MORE team provides a unified clinical review of the program, while the product center makes the final approval determination, considering both clinical and non-clinical information holistically. These include Fast Track Designation, Breakthrough Therapy Designation, Priority Review Designation, and the Accelerated Approval Pathway (AAP).

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New Drug Approvals

MAY 8, 2025

7] Pirtobrutinib was approved for medical use in the United States in January 2023, [4] [8] [9] [10] and in the European Union in November 2023. [2] 7] [12] In the European Union, pirtobrutinib is indicated for the treatment of mantle cell lymphoma. [2] 6] The same trial was used to assess safety and efficacy. [6] 12 (5): 80.

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The Pharma Data

JULY 2, 2025

Food and Drug Administration’s (FDA) accelerated approval of Lynozyfic™ (linvoseltamab-gcpt), a first-in-class BCMAxCD3 bispecific antibody, for the treatment of adults with relapsed or refractory (R/R) multiple myeloma (MM). Lynozyfic demonstrated early, deep, and durable responses in heavily pre-treated patients.

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The Pharma Data

JUNE 15, 2025

Food and Drug Administration (FDA)-approved gene therapy for the treatment of Duchenne muscular dystrophy (DMD). For ambulatory patients — those retaining some ability to walk — there are no new treatment interruptions or regimen modifications at this time. The FDA agrees with this cautious approach.

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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] hdl : 10665/330984. 24 March 2023.

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New Drug Approvals

JUNE 29, 2025

Taletrectinib CAS 1505514-27-1 as salt: 1505515-69-4 , Taletrectinib adipate FDA 6/11/2025, Ibtrozi, To treat locally advanced or metastatic ROS1-positive non-small cell lung cancer AB-106, DS-6051a 405.5 1] Taletrectinib was approved for medical use in the United States in June 2025. [3] 1] Taletrectinib is a kinase inhibitor. [1]

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New Drug Approvals

APRIL 18, 2025

Formula C 27 H 29 Cl 2 FN 2 OS Crinecerfont , sold under the brand name Crenessity , is a medication used for the treatment of congenital adrenal hyperplasia. [1] 2] Crinecerfont was approved for medical use in the United States in December 2024. [2] 2] Crinecerfont was approved for medical use in the United States in December 2024. [2]

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New Drug Approvals

JULY 10, 2025

g/mol O5ZD2TU2B7 FDA 7/3/2025, Ekterly, To treat acute attacks of hereditary angioedema N -[(3-fluoro-4-methoxypyridin-2-yl)methyl]-3-(methoxymethyl)-1-[[4-[(2-oxopyridin-1-yl)methyl]phenyl]methyl]pyrazole-4-carboxamide Sebetralstat , sold under the brand name Ekterly , is a medication used for the treatment of hereditary angioedema. [1]

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New Drug Approvals

JULY 8, 2025

It is being investigated as a potential treatment for various herpes infections, including those resistant to traditional antivirals like acyclovir. Clinical Trials: Pritelivir is currently in phase II clinical trials, with ongoing research into its effectiveness and safety.

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Codon

JUNE 5, 2025

.” Four years earlier, in 2019, Gray had become the first patient with sickle cell anemia — a genetic disorder that causes red blood cells to become sticky and rigid — to receive an experimental treatment using CRISPR genome editing. Dozens more clinical trials, based upon similar gene-editing technologies, are now underway.

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Codon

NOVEMBER 3, 2024

In the 1924 novel, The Magic Mountain , Thomas Mann describes a sanatorium patient named Anton Ferge as he undergoes a painful tuberculosis (TB) treatment. A 1994 review of 14 prospective trials and 12 case-control studies revealed that the BCG vaccine reduced the risk of TB by 50 percent. million people every year.

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The Pharma Data

JULY 2, 2025

FDA Grants Priority Review to Merck’s WINREVAIR™ Based on Landmark ZENITH Trial Showing Dramatic Reduction in Morbidity and Mortality in PAH Patients Merck (NYSE: MRK), operating as MSD outside the U.S. label of WINREVAIR based on compelling new evidence from the Phase 3 ZENITH trial. risk score of 9 or greater.

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New Drug Approvals

APRIL 19, 2025

Landiolol 133242-30-5 ONO-1101 Ono 1101 WHO 7516 FDA APPROVED 11/22/2024, Rapiblyk , To treat supraventricular tachycardia C25H39N3O8 509.6 Landiolol 133242-30-5 ONO-1101 Ono 1101 WHO 7516 FDA APPROVED 11/22/2024, Rapiblyk , To treat supraventricular tachycardia C25H39N3O8 509.6 9] It is used as landiolol hydrochloride.

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The Pharma Data

JUNE 13, 2025

FDA Approves KEYTRUDA® (Pembrolizumab) for Perioperative Treatment of Resectable Locally Advanced Head and Neck Squamous Cell Carcinoma Merck known as MSD outside the United States and Canada, recently announced that the U.S. Importantly, these side effects may manifest during or after treatment with KEYTRUDA.

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Codon

NOVEMBER 1, 2024

Amodei also imagines the ways AI could accelerate biological research and yield miraculous cures in the 21st century; everything from the prevention and treatment of nearly all infectious and inherited diseases to the elimination of most cancers. Human constraints also play a role at a later stage, when clinical trials are involved.”

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The Pharma Data

JUNE 11, 2025

FDA Expands Approval of AbbVie’s MAVYRET® as First and Only 8-Week Treatment for Acute Hepatitis C in Adults and Children Aged 3 and Above AbbVie has received a significant regulatory boost for its hepatitis C treatment portfolio as the U.S. Safety outcomes from the trial further bolster the confidence in MAVYRET’s use.

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Drug Patent Watch

JANUARY 7, 2025

As healthcare professionals, it’s our responsibility to educate patients about generic drugs and empower them to make informed decisions about their treatment options. Explain the FDA Approval Process Many patients are unaware of the rigorous approval process generic drugs must undergo.

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FDA Law Blog: Drug Discovery

JUNE 18, 2025

As defined by the National Cancer Institute, tumor board meetings convene multidisciplinary specialists to decide on the best treatment plan for new and complex cancer cases. FDA plans to issue a limited number of vouchers, without specifying an exact number as of yet. Easily interpreted clinical trial endpoints (e.g.,

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Drugs.com

FEBRUARY 5, 2025

Food and Drug Administration has approved Symbravo (meloxicam and rizatriptan) for the acute treatment of migraine with or without aura in adults. The approval was based on the results of three phase 3 trials of. WEDNESDAY, Feb. 5, 2025 -- The U.S.

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BioPharma Drive: Drug Pricing

JUNE 4, 2024

A group of independent experts wasn't convinced by clinical trial data from company Lykos Therapeutics, which is seeking FDA approval of MDMA-assisted treatment for post-traumatic stress disorder.

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PLOS: DNA Science

MARCH 28, 2024

FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. The New Drug Duvyzat (givinostat), a type of drug called an HDAC inhibitor, has been in clinical trials to treat cancers and other disorders of the blood, Crohn’s disease, and a form of juvenile arthritis.

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