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Keytruda – KN-158 for small cell lung cancer (SCLC). PDUFA date under priority review June 17, 2019.
The FDA has accepted and granted priority review for a new supplemental Biologics License Application (sBLA) for KEYTRUDA, Merck’s anti-PD-1 therapy, as monotherapy for the treatment of patients with advanced small cell lung cancer (SCLC) whose disease has progressed after two or more lines of prior therapy. This sBLA, which is seeking accelerated approval for this new indication, is based on data from the SCLC cohorts of the Phase 2 KEYNOTE-158 and Phase 1b KEYNOTE-028 trials. The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of June 17, 2019.
“There is a significant need for new treatment options for small cell lung cancer, which has a five-year survival rate of only six percent overall,” said Dr. Jonathan Cheng, vice president, oncology clinical research, Merck Research Laboratories. “KEYTRUDA has already been established as an important treatment option for many patients with advanced non-small cell lung cancer and this acceptance provides an opportunity to potentially benefit even more patients.”
This acceptance marks the first U.S. application for KEYTRUDA in SCLC and demonstrates Merck’s commitment to bringing forward new treatment options for patients with historically difficult-to-treat cancers, like SCLC. As part of our broad clinical development in lung cancer, KEYTRUDA is being studied in combination with chemotherapy in the ongoing Phase 3 KEYNOTE-604 study in patients with newly diagnosed extensive stage SCLC.
Lung cancer, which forms in the tissues of the lungs, usually within cells lining the air passages, is the leading cause of cancer death worldwide. Each year, more people die of lung cancer than die of colon and breast cancers combined. The two main types of lung cancer are non-small cell and small cell. Small cell lung cancer (SCLC) accounts for about 10 to 15 percent of all lung cancers. The five-year survival rate for patients diagnosed in the United States with any stage of SCLC is estimated to be six percent.
KEYTRUDA and Inlyta - KEYNOTE-426 for renal cell carcinoma. PDUFA date under priority review June 20, 2019. The FDA has accepted and granted priority review for a new supplemental Biologics License
KEYTRUDA and Inlyta – KEYNOTE-426 for renal cell carcinoma. PDUFA date under priority review June 20, 2019.
The FDA has accepted and granted priority review for a new supplemental Biologics License Application (sBLA) for KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with Inlyta (axitinib), a tyrosine kinase inhibitor, for the first-line treatment of patients with advanced renal cell carcinoma (RCC). This sBLA is based on findings from the Phase 3 KEYNOTE-426 trial, which demonstrated that KEYTRUDA in combination with axitinib, as compared to sunitinib, significantly improved overall survival (OS) and progression-free-survival (PFS) in the first-line treatment of advanced RCC. These data will be presented at the 2019 Genitourinary Cancers Symposium (ASCO GU) in San Francisco on February 16. The sBLA also included supporting data from the Phase 1b KEYNOTE-035 trial. The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of June 20, 2019.
“Many patients with advanced renal cell carcinoma face a poor prognosis and there remains a need for new and effective treatment options in the first-line setting,” said Dr. Roger M. Perlmutter, president, Merck Research Laboratories. “KEYNOTE-426 demonstrated that an anti-PD-1 combination therapy significantly improved overall survival and progression-free survival versus sunitinib in the first-line treatment of advanced renal cell carcinoma. We look forward to working with the FDA to bring this KEYTRUDA combination to patients.”
Merck has filed data from KEYNOTE-426 with regulatory authorities worldwide. Merck has an extensive clinical development program across clear cell and non-clear cell RCC and is advancing multiple potential registration-enabling studies with KEYTRUDA, as monotherapy and in combination with other treatments, including KEYNOTE-564 and KEYNOTE-581.
Renal cell carcinoma (RCC) is by far the most common type of kidney cancer; about 9 out of 10 kidney cancers are renal cell carcinomas. Renal cell carcinoma is about twice as common in men as in women. Modifiable risk factors include smoking, obesity, workplace exposure to certain substances and high blood pressure. There were approximately 403,000 cases of kidney cancer diagnosed worldwide in 2018 and about 175,000 deaths from the disease. In the U.S. alone, there will be an estimated 74,000 new cases of kidney cancer diagnosed in 2019 and about 15,000 people will die from the disease.
KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.
Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 900 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient’s likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Ivosidenib for frontline AML with IDH1 mutation. PDUFA date under priority review June 21, 2019. The FDA has accepted the company’s supplemental New Drug Application (sNDA) for TIBSOVO® (ivosidenib) for the
Ivosidenib for frontline AML with IDH1 mutation. PDUFA date under priority review June 21, 2019.
The FDA has accepted the company’s supplemental New Drug Application (sNDA) for TIBSOVO® (ivosidenib) for the treatment of patients with newly diagnosed acute myeloid leukemia (AML) with an isocitrate dehydrogenase 1 (IDH1) mutation who are not eligible for standard therapy.
The sNDA was granted Priority Review and has been given a Prescription Drug User Fee Act (PDUFA) action date of June 21, 2019. The FDA’s Priority Review status accelerates the review time from 10 months to a goal of six months from the day of filing acceptance and is given to drugs that may offer major advances in treatment or may provide a treatment where no adequate therapy exists. In addition, the FDA accepted the TIBSOVO® sNDA under its Real-Time Oncology Review pilot program, which aims to make the review of oncology drugs more efficient by allowing the FDA access to clinical trial data before the information is formally submitted to the agency.
“In less than seven months since TIBSOVO’s approval in relapsed or refractory AML, we are pleased to be working with the FDA to expand its labeled indication into the frontline setting,” said Chris Bowden, M.D., chief medical officer of Agios. “Patients with newly diagnosed AML who are not eligible for standard treatments, such as intensive and non-intensive chemotherapy, are currently offered only palliative care. There is tremendous need for new treatment options, and we believe AML patients with IDH1 mutations have the potential to benefit from this targeted therapy.”
TIBSOVO® is a first-in-class, oral, targeted inhibitor of mutant IDH1. The sNDA submission is based on results from the untreated AML patients from the Phase 1 dose-escalation and expansion study of ivosidenib in patients with newly diagnosed AML ineligible for standard treatment. Data from the untreated AML portion of this study were presented at the 2018 American Society of Hematology (ASH) Annual Meeting.
Agios Pharmaceuticals News
Vyleesi (Bremelanotide) for female sexual dysfunction (FSD). PDUFA date extended to June 23, 2019. AMAG
EDSIVO for vascular Ehlers-Danlos Syndrome. PDUFA date under priority review June 25, 2019. Acer Therapeutics announced back on December 26, 2018, that the FDA had accepted for review Acer’s New Drug
EDSIVO for vascular Ehlers-Danlos Syndrome. PDUFA date under priority review June 25, 2019.
Acer Therapeutics announced back on December 26, 2018, that the FDA had accepted for review Acer’s New Drug Application (NDA) for EDSIVO™ for the treatment of vascular Ehlers-Danlos syndrome (vEDS) in patients with a confirmed type III collagen (COL3A1) mutation. The FDA also granted a priority review of the NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 25, 2019. Priority review is a designation granted by the FDA to accelerate the review process for drugs that offer a significant improvement in treatment or provide treatment where no satisfactory alternative therapy exists.
“The acceptance of our NDA for EDSIVO™ is an important step in our efforts to help patients with vEDS, who suffer with a devastating disease that currently has no approved treatment,” said William Andrews, M.D., FACP, Chief Medical Officer of Acer. “We have had the honor of learning about the significant challenges of living with vEDS directly from patients and their families. This has in large part driven the hard work, passion and complete dedication that our small team has given to this effort, and we will continue to do so as the FDA reviews our NDA for EDSIVO™. We are excited about the possibility of making EDSIVO™ available in the U.S. for patients in the near future.”
“We continue to accelerate our pre-commercial activities supporting the potential U.S. launch of EDSIVO™ for the treatment of vEDS if it is approved by the FDA,” said Chris Schelling, CEO and Founder of Acer. “Additionally, we are working diligently on advancing and expanding our pipeline with the goal of bringing multiple products to patients with serious rare diseases over the next several years.”
Ehlers-Danlos Syndrome (EDS) is a group of hereditary disorders of connective tissue. vEDS is the most severe subtype where patients suffer from life threatening arterial dissections and ruptures, as well as intestinal and uterine ruptures. The average mortality is 51 years of age. An Acer-commissioned patient-finder study phenotypically identified 4,169 vEDS patients in the U.S. from an analysis of a commercially available patient claims database with data of approximately 190 million unique patient lives. Based on that information, Acer estimates the prevalence of phenotypically-defined vEDS in the U.S. could be greater than 1 in 45,000. Currently, there are no FDA-approved therapies for vEDS. Acer is advancing EDSIVO™ (celiprolol), a new chemical entity (NCE), for the treatment of vEDS based on a randomized controlled clinical study of celiprolol(1). FDA granted a priority review of the EDSIVO™ NDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 25, 2019. EDSIVO™ received FDA Orphan Drug Designation for the potential treatment of vEDS in 2015.
Acer, headquartered in Newton, MA, is a pharmaceutical company focused on the acquisition, development and commercialization of therapies for patients with serious rare and ultra-rare diseases with critical unmet medical need. Acer’s late-stage clinical pipeline includes two candidates for severe genetic disorders: EDSIVO™ (celiprolol) for vascular Ehlers-Danlos syndrome (vEDS), and ACER-001 (a fully taste-masked, immediate release formulation of sodium phenylbutyrate) for urea cycle disorders (UCD) and Maple Syrup Urine Disease (MSUD). There are no FDA-approved drugs for vEDS and MSUD and limited options for UCD, which collectively impact approximately 7,000 patients in the U.S. Acer’s product candidates have clinical proof-of-concept and mechanistic differentiation, and Acer intends to seek approval for them in the U.S. by using the regulatory pathway established under section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act (FFDCA) that allows an applicant to rely at least in part on third-party data for approval, which may expedite the preparation, submission, and approval of a marketing application.
Acer Therapeutics News
UPDATE: Dupixent (dupilumab) for atopic dermatitis 12-17 year-olds. PDUFA date under priority review June 26, 2019. The FDA has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent®
UPDATE: Dupixent (dupilumab) for atopic dermatitis 12-17 year-olds. PDUFA date under priority review June 26, 2019.
The FDA has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Dupixent® (dupilumab) in adolescent patients 12 to 17 years of age with moderate-to-severe atopic dermatitis, whose disease was inadequately controlled with topical therapies or for whom topical treatment was medically inadvisable. Currently, there are no FDA-approved systemic biologic medicines to treat adolescents with moderate-to-severe atopic dermatitis. The target action date for the FDA decision is June 26, 2019.
The sBLA is supported by data from a pivotal Phase 3 trial evaluating the efficacy and safety of Dupixent monotherapy in adolescent patients with moderate-to-severe atopic dermatitis, which were presented at the European Academy of Dermatology and Venereology in September 2018.
Dupixent works by inhibiting interleukin-4 and interleukin-13 (IL-4 and IL-13) signaling, which is one of the important contributors to Type 2 inflammation, a systemic response known to play a role in moderate-to-severe atopic dermatitis.
Dupixent is currently approved in the U.S. as a treatment for adults with moderate-to-severe atopic dermatitis whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable; and as add-on maintenance treatment for patients 12 years and older with moderate-to-severe asthma with an eosinophilic phenotype or with oral corticosteroid-dependent asthma. In 2016, the FDA granted Breakthrough Therapy designation for Dupixent for the treatment of moderate-to-severe (adolescents 12 to 17 years of age) and severe (children 6 months to 11 years of age) atopic dermatitis not well controlled on topical prescription medications.
Dupixent is also approved for use in certain adult patients with moderate-to-severe atopic dermatitis in countries of the European Union, and other countries including Canada and Japan. In the U.S., more than 60,000 adult patients with atopic dermatitis have been prescribed Dupixent to date.
Regeneron Pharmaceuticals News
REVLIMID - AUGMENT for Relapsed or Refractory Follicular Lymphoma. PDUFA date under priority review June 27, 2019. The FDA has granted Priority Review designation for the company’s supplemental New Drug Application
REVLIMID – AUGMENT for Relapsed or Refractory Follicular Lymphoma. PDUFA date under priority review June 27, 2019.
The FDA has granted Priority Review designation for the company’s supplemental New Drug Application (sNDA) for REVLIMID® (lenalidomide) in combination with rituximab (R²) for the treatment of patients with previously treated follicular and marginal zone lymphoma. Under the Prescription Drug User Fee Act (PDUFA), the FDA has set its action date as June 27, 2019.
“R2 has the potential to offer patients with previously treated follicular lymphoma and marginal zone lymphoma a chemotherapy free option” said Jay Backstrom, M.D., Chief Medical Officer and Head of Global Regulatory Affairs for Celgene. “We look forward to working with the FDA to bring the R2 regimen to patients as quickly as possible.”
The sNDA is based on results from the randomized, double-blind, phase 3 AUGMENT study, which evaluated the efficacy and safety of the investigational R² combination versus rituximab plus placebo in patients with relapsed/refractory follicular and marginal zone lymphoma. Results from the study were presented at the 2018 American Society of Hematology (ASH) Annual Meeting and Exposition.
Earlier this year, Celgene submitted and had accepted a Marketing Authorization Application (MAA) for R2 to the European Medicines Agency (EMA) for the treatment of relapsed/refractory follicular and marginal zone lymphoma.
REVLIMID alone or in combination with other agents is not approved for use in follicular lymphoma or marginal zone lymphoma in any geography.
Celgene also announced updated timing for the anticipated submission of a Biologics License Application (BLA) with the U.S. FDA for luspatercept in adult patients with anemia related to very low to intermediate myelodysplastic syndromes (MDS) with ring sideroblasts who require red blood cell transfusions, and in adult patients with anemia related to beta-thalassemia who require regular red blood cell transfusions. The company expects to submit the BLA in April 2019.
Luspatercept is not approved in any region for any indication. Acceleron Pharma Inc. and Celgene are jointly developing luspatercept as part of a global collaboration.
Lymphoma is a blood cancer that develops in lymphocytes, a type of white blood cell in the immune system that helps protect the body from infection. There are two classes of lymphoma – Hodgkin’s and non-Hodgkin’s lymphoma (NHL) – each with specific subtypes that determine how the cancer behaves, spreads and should be treated.
Indolent lymphomas are slow-growing forms of the disease, which can often be asymptomatic or have fewer symptoms upon diagnosis. Indolent lymphomas account for approximately 40 percent of all NHL cases. In patients with relapsed/refractory lymphoma, the disease has either responded to treatment but then returned or has not responded to initial treatment.
Eculizumab for relapsing Neuromyelitis Optica Spectrum Disorder (NMOSD). PDUFA date under priority review June 28, 2019. The FDA has accepted for review a supplemental Biologics License Application (sBLA) for the use
Eculizumab for relapsing Neuromyelitis Optica Spectrum Disorder (NMOSD). PDUFA date under priority review June 28, 2019.
The FDA has accepted for review a supplemental Biologics License Application (sBLA) for the use of SOLIRIS® (eculizumab), the company’s first C5 complement inhibitor, as a treatment for patients with neuromyelitis optica spectrum disorder (NMOSD) who have anti-aquaporin-4 (AQP4) auto antibodies. The FDA granted priority review and set a Prescription Drug User Fee Act (PDUFA) action date of June 28, 2019. The application is supported by comprehensive data from the successful PREVENT study in patients with anti-AQP4 auto antibody-positive NMOSD.
“Given the debilitating impact NMOSD relapses can have on patients and the fact that there is currently no approved therapy, we are committed to getting SOLIRIS to these patients as quickly as possible,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “Based on our strong clinical data, we believe that SOLIRIS can provide significant therapeutic benefits, and we look forward to working with the FDA to facilitate a rapid review.”
NMOSD is a rare, devastating, complement-mediated disorder of the central nervous system characterized by relapses. Each relapse results in stepwise accumulation of disability, including blindness and paralysis, and sometimes premature death.1,2,3 Patients who have anti-AQP4 auto-antibodies represent approximately three quarters of all patients with NMOSD.
The European Medicines Agency (EMA) is reviewing Alexion’s application to add the treatment of NMOSD to the marketing authorization for SOLIRIS in the European Union (EU), submitted in January 2019. In addition, Alexion is preparing to submit a supplemental New Drug Application for SOLIRIS in NMOSD in Japan in Q1 2019. SOLIRIS has received Orphan Drug Designation (ODD) for the treatment of patients with NMOSD in the U.S., EU and Japan.
Alexion Pharmaceuticals News
Avatrombopag for immune Thrombocytopenic Purpura (ITP). PDUFA date for sNDA filing June 30, 2019. The FDA has accepted for review the supplemental New Drug Application (sNDA) for DOPTELET (avatrombopag) for a
Avatrombopag for immune Thrombocytopenic Purpura (ITP). PDUFA date for sNDA filing June 30, 2019.
The FDA has accepted for review the supplemental New Drug Application (sNDA) for DOPTELET (avatrombopag) for a new indication, the treatment of chronic immune thrombocytopenia (ITP) in patients who have had an insufficient response to a previous treatment. ITP is an autoimmune bleeding disorder characterized by thrombocytopenia, i.e., an abnormally low level of platelets. The Prescription Drug User Fee Act (PDUFA) goal date for an FDA decision on the sNDA is June 30, 2019.
DOPTELET, a second generation, orally administered thrombopoietin receptor agonist (TPO-RA), was approved by FDA in May 2018 for the treatment of thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure.
“Chronic ITP affects approximately 60,000 adults in the United States and despite the currently available therapies, which include two other TPO-RAs, there remains an important unmet need,” said Lee F. Allen, MD, PhD and Chief Medical Officer of Dova. “Acceptance of this sNDA is another significant milestone for Dova, and an important step towards addressing this underserved patient population and expanding the applications for DOPTELET as a treatment for thrombocytopenia. We look forward to working closely with the FDA as they review this sNDA.”
The ITP sNDA is supported by safety and efficacy data from one pivotal randomized, placebo-controlled Phase 3 clinical trial in the target indication that met its primary (number of weeks with a platelet count ≥50×109/L in the absence of rescue therapy) and first secondary (proportion of subjects with platelet counts ≥50×109/L on Day 8) efficacy endpoints with high statistical significance (P