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U.S. Tuberous Sclerosis Drug Market Projected to Reach $1.25 Billion by 2030, Growing at 23.1% CAGR

Drug ApprovalNeurologyRare Disease
  • The U.S. tuberous sclerosis drug market is expected to grow from $237.2 million in 2022 to $1.25 billion by 2030, driven by FDA approvals of new treatments and increasing disease prevalence.
  • FDA-approved treatments include Afinitor (everolimus) for patients aged 2 and above, approved in 2018, and Epidiolex (cannabidiol) for patients aged 1 and above, approved in 2020.
  • mTOR inhibitors are gaining significant traction in treatment protocols, showing effectiveness for internal tumors and skin lesions, with FDA approval for treating lung complications like Lymphangioleiomyomatosis (LAM) in 2015.

USDA Approves First Therapeutic Treatment for Canine Parvovirus Using Monoclonal Antibody Technology

Monoclonal AntibodyDrug Approval
  • The USDA has granted conditional license for Elanco's Canine Parvovirus Monoclonal Antibody, marking the first approved therapeutic solution to treat canine parvovirus.
  • In efficacy studies, all 28 treated puppies survived with significantly faster recovery times for vomiting, inappetence, and lethargy compared to traditional supportive care.
  • The single intravenous dose treatment could reduce hospitalization costs and make parvovirus treatment more accessible to pet owners who previously couldn't afford intensive care.
  • This breakthrough represents the first monoclonal antibody treatment for Elanco and addresses a critical unmet need in veterinary medicine for a disease with over 90% fatality rate if untreated.

Novel BCMA-Targeted Therapies and Emerging Agents Transform Treatment Landscape for Relapsed/Refractory Multiple Myeloma

CAR-TDrug Approval
  • BCMA-directed therapies including CAR T-cell products ide-cel and cilta-cel, along with bispecific T-cell engager teclistamab, have demonstrated unprecedented efficacy in heavily pretreated patients with relapsed/refractory multiple myeloma.
  • Emerging bispecific antibodies elranatamab and talquetamab show promising response rates of 61-74% in triple-class refractory patients, offering new treatment options for those who have exhausted conventional therapies.
  • Novel agents including mezigdomide (a CELMoD), purinostat mesylate (HDAC inhibitor), and bispecific CS1-BCMA CAR T cells are expanding therapeutic options with manageable safety profiles.
  • The treatment paradigm is shifting toward earlier use of BCMA-targeted therapies and novel combinations, as patients increasingly become refractory to multiple drug classes including immunomodulators, proteasome inhibitors, and anti-CD38 antibodies.

Isatuximab, Pomalidomide, Elotuzumab and Dexamethasone in Relapsed And/or Refractory Multiple Myeloma

NCT04835129RecruitingPhase 2
Medical College of Wisconsin
Posted 1/10/2022

Selinexor and Backbone Treatments of Multiple Myeloma Patients

NCT02343042Active, Not RecruitingPhase 1
Karyopharm Therapeutics Inc
Posted 10/1/2015

Trastuzumab Deruxtecan Gains Regulatory Approvals in China and Europe for HER2-Positive Cancers

Drug ApprovalMonoclonal Antibody
  • China's National Medical Products Administration approved trastuzumab deruxtecan for HER2-positive breast cancer patients who received prior anti-HER2 therapies, based on DESTINY-Breast03 trial showing 72% reduction in disease progression risk.
  • The European Commission approved trastuzumab deruxtecan as second-line treatment for HER2-positive advanced gastric cancer, marking the first antibody-drug conjugate approved in Europe for this indication.
  • DESTINY-Breast03 demonstrated superior efficacy with median progression-free survival not reached for trastuzumab deruxtecan versus 6.8 months for trastuzumab emtansine.
  • The approvals expand treatment options for patients with HER2-positive cancers who face poor outcomes after initial therapy progression.

Trastuzumab Deruxtecan (DS-8201a) Versus Investigator's Choice for HER2-low Breast Cancer That Has Spread or Cannot be Surgically Removed [DESTINY-Breast04]

NCT03734029Active, Not RecruitingPhase 3
Daiichi Sankyo
Posted 12/27/2018

FDA Approves Elacestrant as First Oral SERD for Advanced ER+/HER2- Breast Cancer

Drug Approval
  • The FDA has approved elacestrant (Orserdu), marking the first oral selective estrogen receptor degrader (SERD) to demonstrate improved efficacy over standard of care treatments in advanced breast cancer.
  • In the phase 3 EMERALD trial, elacestrant reduced the risk of progression or death by 30% in all patients and by 45% in patients with ESR1 mutations compared to standard endocrine therapy.
  • The approval provides a new oral treatment option for patients with estrogen receptor-positive/HER2-negative advanced or metastatic breast cancer following prior endocrine therapy including CDK4/6 inhibitors.
  • Elacestrant showed manageable safety profile with nausea being the most common adverse event, occurring in 35% of patients in the treatment arm.

ELACESTRANT in Women and Men With CDK4/6 Inhibitor-Naive Estrogen Receptor Positive, HER-2 Negative Metastatic Breast Cancer Study

NCT05596409Active, Not RecruitingPhase 2
Stemline Therapeutics, Inc.
Posted 5/19/2023

Study of Efficacy of Ribociclib After Progression on CDK4/6 Inhibition in Patients With HR+ HER2- Advanced Breast Cancer

NCT02632045CompletedPhase 2
Melissa K Accordino
Posted 3/1/2016

A Study Evaluating the Efficacy and Safety of Giredestrant Compared With Physician's Choice of Endocrine Monotherapy in Participants With Previously Treated Estrogen Receptor-Positive, HER2-Negative Locally Advanced or Metastatic Breast Cancer (acelERA Breast Cancer)

NCT04576455Active, Not RecruitingPhase 2
Hoffmann-La Roche
Posted 11/27/2020

Open-Label Umbrella Study To Evaluate Safety And Efficacy Of Elacestrant In Various Combination In Patients With Metastatic Breast Cancer

NCT05563220RecruitingPhase 1
Stemline Therapeutics, Inc.
Posted 1/24/2023

Phase 3 Trial of Elacestrant vs. Standard of Care for the Treatment of Patients With ER+/HER2- Advanced Breast Cancer

NCT03778931CompletedPhase 3
Stemline Therapeutics, Inc.
Posted 5/10/2019

A Study of Imlunestrant, Investigator's Choice of Endocrine Therapy, and Imlunestrant Plus Abemaciclib in Participants With ER+, HER2- Advanced Breast Cancer

NCT04975308Active, Not RecruitingPhase 3
Eli Lilly and Company
Posted 10/4/2021

A Study to Investigate Efficacy and Safety With Oral AZD9833 Compared With Intramuscular Fulvestrant in Post-menopausal Women at Least 18 Years of Age With Advanced ER-positive HER2 Negative Breast Cancer

NCT04214288Active, Not RecruitingPhase 2
AstraZeneca
Posted 4/22/2020

Study of Abemaciclib and Elacestrant in Patients With Brain Metastasis Due to ER+/HER-2- Breast Cancer

NCT05386108RecruitingPhase 1
Stemline Therapeutics, Inc.
Posted 8/31/2022

Phase 2 Study of Amcenestrant (SAR439859) Versus Physician's Choice in Locally Advanced or Metastatic ER-positive Breast Cancer

NCT04059484TerminatedPhase 2
Sanofi
Posted 10/22/2019

FDA Authorizes Novavax's Protein-Based COVID-19 Vaccine for US Adults

Drug Approval
• The US Food and Drug Administration has authorized Novavax's protein-based COVID-19 vaccine for adults, making it the fourth COVID-19 shot available in the United States.
• Novavax's vaccine uses traditional protein-based technology that has been employed for decades against diseases like hepatitis B and influenza, potentially appealing to the 27 million unvaccinated American adults skeptical of mRNA platforms.
• The US government has secured 3.2 million Novavax doses to be released after quality testing, though European uptake has been limited with only about 242,000 doses administered since its December launch.

Eagle Pharmaceuticals Submits NDA for Landiolol, Ultra-Short-Acting Beta Blocker for Critical Care Tachycardia

Drug Approval
  • Eagle Pharmaceuticals announced that AOP Orphan Pharmaceuticals submitted a new drug application to the FDA for landiolol, seeking approval for short-term reduction of ventricular rate in patients with supraventricular tachycardia.
  • Landiolol is an ultra-short-acting, cardio-selective beta-1 adrenergic blocker with a β1:β2 ratio of 255:1, making it the most cardioselective beta blocker available.
  • The drug is already commercially available in Japan and several European markets, with multiple clinical studies demonstrating its safety and efficacy for rapid short-term control of tachyarrhythmias.
  • If approved, Eagle expects five years of new chemical entity exclusivity and anticipates approval by mid-year 2023 based on FDA feedback from a Type C meeting.

Eskayef Pharmaceuticals Becomes Third Bangladeshi Company to Receive US FDA Approval for Manufacturing Facility

Drug Approval
  • Eskayef Pharmaceuticals Ltd received US FDA approval for its solid manufacturing facility in Tongi, Gazipur, becoming the third Bangladeshi pharmaceutical company to achieve this milestone after Square and Beximco Pharma.
  • The approval enables Eskayef to export Pregabalin capsules in eight different strengths (25mg to 300mg) to the US market, with the anti-epileptic and neuropathic pain medication set to launch shortly.
  • This achievement follows more than five years of efforts and represents a significant step toward Bangladesh's pharmaceutical export growth, which reached $169 million in 2020-21, more than doubling from $83 million five years earlier.
  • The FDA approval validates Eskayef's manufacturing quality standards and opens doors to the world's largest pharmaceutical market, reinforcing the company's global presence across 67 countries.

Moderna's COVID-19 Vaccine Spikevax Receives Full FDA Approval, Marking Company's First Licensed Product

Drug Approval
  • Moderna's COVID-19 vaccine Spikevax received full FDA approval for individuals 18 years and older, becoming the company's first licensed product in the United States.
  • The approval was based on clinical trial data showing 93% efficacy in preventing COVID-19 and 98% effectiveness in preventing severe disease among 28,451 participants.
  • Spikevax has now received regulatory approval in more than 70 countries, with Moderna having shipped 807 million doses globally in 2021.
  • The vaccine maintains the same formulation as the emergency-use authorization version and is administered as a two-dose primary series one month apart.

Polatuzumab Vedotin Shows Superior Efficacy in First-Line DLBCL Treatment and Confirms Long-Term Benefits in Relapsed Disease

Monoclonal AntibodyDrug Approval
  • The phase 3 POLARIX trial demonstrated that polatuzumab vedotin plus R-CHP (pola-R-CHP) significantly improved progression-free survival compared to standard R-CHOP in previously untreated DLBCL patients, reducing the risk of disease progression by 27%.
  • Long-term follow-up data from the GO29365 study confirmed durable disease control with polatuzumab vedotin plus bendamustine and rituximab in relapsed/refractory DLBCL, with 25% of patients achieving ongoing responses lasting over 2 years.
  • Both studies showed manageable safety profiles for polatuzumab vedotin combinations, with peripheral neuropathy rates comparable to standard regimens and no new safety signals identified with extended follow-up.

Polatuzumab Vedotin, Rituximab, Ifosfamide, Carboplatin, and Etoposide (PolaR-ICE) as Initial Salvage Therapy for the Treatment of Relapsed/Refractory Diffuse Large B-Cell Lymphoma

NCT04665765Active, Not RecruitingPhase 2
City of Hope Medical Center
Posted 1/18/2021

A Randomized, Multicenter, Phase III Trial Comparing Treatment With R-mini-CHOP With R-mini-CHP + Polatuzumab Vedotin in Patients With Diffuse Large Cell B Cell Lymphoma

NCT04332822RecruitingPhase 3
Nordic Lymphoma Group
Posted 8/19/2020

A Study of Polatuzumab Vedotin (DCDS4501A) in Combination With Rituximab or Obinutuzumab Plus Bendamustine in Participants With Relapsed or Refractory Follicular or Diffuse Large B-Cell Lymphoma

NCT02257567CompletedPhase 1
Hoffmann-La Roche
Posted 10/15/2014

A Study Comparing the Efficacy and Safety of Polatuzumab Vedotin With Rituximab-Cyclophosphamide, Doxorubicin, and Prednisone (R-CHP) Versus Rituximab-Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone (R-CHOP) in Participants With Diffuse Large B-Cell Lymphoma

NCT03274492Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 11/16/2017

A Study to Evaluate the Safety and Efficacy of Polatuzumab Vedotin in Combination With Rituximab, Gemcitabine and Oxaliplatin Compared to Rituximab, Gemcitabine and Oxaliplatin Alone in Participants With Relapsed or Refractory Diffuse Large B-Cell Lymphoma

NCT04182204CompletedPhase 3
Hoffmann-La Roche
Posted 2/7/2020
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