Orphan drugs

Eisai: FDA Approves LENVIMA (lenvatinib) Plus KEYTRUDA (pembrolizumab) Combination for Patients With Certain Types of Advanced Endometrial Carcinoma

Retrieved on: 
Friday, July 23, 2021

Based on the type and/or severity of the adverse reaction, LENVIMA may be interrupted, reduced and/or discontinued.

Key Points: 
  • Based on the type and/or severity of the adverse reaction, LENVIMA may be interrupted, reduced and/or discontinued.
  • Based on its mechanism of action and data from animal reproduction studies, LENVIMA can cause fetal harm when administered to a pregnant woman.
  • Based on the severity of the adverse reaction, KEYTRUDA should be withheld or permanently discontinued and corticosteroids administered if appropriate.
  • "Based on Phase 3 data, today's approval acts as the confirmatory trial to our previous accelerated approval of KEYTRUDA plus LENVIMA in patients with certain types of advanced endometrial cancer and reinforces the impact of our joint research with Eisai in exploring the potential of this combination to treat more patients with challenging types of cancer."

Exelixis to Release Second Quarter 2021 Financial Results on Thursday, August 5, 2021

Retrieved on: 
Thursday, July 22, 2021

Exelixis, Inc. (Nasdaq: EXEL) announced today that its second quarter 2021 financial results will be released on Thursday, August 5, 2021 after the markets close.

Key Points: 
  • Exelixis, Inc. (Nasdaq: EXEL) announced today that its second quarter 2021 financial results will be released on Thursday, August 5, 2021 after the markets close.
  • At 5:00 p.m. EDT / 2:00 p.m. PDT, Exelixis management will host a conference call and webcast to discuss the results and provide a general business update.
  • For more information about Exelixis, please visit www.exelixis.com , follow @ExelixisInc on Twitter or like Exelixis, Inc. on Facebook.
  • Exelixis, the Exelixis logo, CABOMETYX, COMETRIQ and COTELLIC are registered trademarks of Exelixis, Inc. MINNEBRO is a registered trademark of Daiichi Sankyo Company, Limited.

Radius Health Announces Plans for Global Prader-Willi Syndrome Pivotal Study

Retrieved on: 
Thursday, July 22, 2021

Based on the feedback received, Radius plans on initiating a pivotal Phase 2/3 global study for patients with PWS.

Key Points: 
  • Based on the feedback received, Radius plans on initiating a pivotal Phase 2/3 global study for patients with PWS.
  • Radius plans to initiate the pivotal Phase 2/3 study, to be branded as SCOUT (Synthetic Cannabidiol Oral Solution), by end of this year or early first quarter of 2022.
  • With this current initiation timeline, it is anticipated top line results would be available in the second half of 2024.
  • Radius is initially targeting Prader-Willi syndrome (PWS) and anticipates initiating a seamless pivotal Phase 2/3 study for patients with PWS in the second half of 2021.

Trial Launches to Evaluate GlycoMimetics’ Uproleselan Added to Cladribine Plus Low-Dose Cytarabine in AML Patients

Retrieved on: 
Thursday, July 22, 2021

GlycoMimetics, Inc. (Nasdaq: GLYC) announced today that clinicians have treated the first patient in a Phase 1b/2 study evaluating the companys lead drug candidate, uproleselan, added to cladribine plus low dose cytarabine (LDAC) in patients with treated secondary AML (ts-AML).

Key Points: 
  • GlycoMimetics, Inc. (Nasdaq: GLYC) announced today that clinicians have treated the first patient in a Phase 1b/2 study evaluating the companys lead drug candidate, uproleselan, added to cladribine plus low dose cytarabine (LDAC) in patients with treated secondary AML (ts-AML).
  • According to Eric Feldman, M.D., GlycoMimetics Chief Medical Officer, Patients with treated secondary AML have an extremely poor prognosis.
  • Our previous preclinical and clinical research supports the potential for these patients to benefit from the addition of uproleselan.
  • The Phase 1b/2 single-arm trial is enrolling patients 18 years or older, with a diagnosis of ts-AML who have not received therapy for their AML.

FDA Approves KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib) Combination for Patients With Certain Types of Advanced Endometrial Carcinoma

Retrieved on: 
Thursday, July 22, 2021

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib.

Key Points: 
  • KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib.
  • KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC).
  • Of the patients who developed pneumonitis, 42% of these patients interrupted KEYTRUDA, 68% discontinued KEYTRUDA, and 77% had resolution.
  • In KEYNOTE-775, when KEYTRUDA was administered in combination with LENVIMA to patients with advanced endometrial carcinoma that were not MSI-H or dMMR (n=342), fatal adverse reactions occurred in 4.7% of patients.

Aprea Therapeutics Announces Positive Results from Phase 2 Trial of Eprenetapopt + Azacitidine for Post-Transplant Maintenance Therapy in TP53 Mutant MDS and AML

Retrieved on: 
Wednesday, July 21, 2021

In addition, the post- transplant regimen of eprenetapopt and azacitidine was well tolerated among patients in the clinical trial.

Key Points: 
  • In addition, the post- transplant regimen of eprenetapopt and azacitidine was well tolerated among patients in the clinical trial.
  • The post-transplant RFS and OS data with eprenetapopt and azacitidine maintenance therapy in these very difficult-to-treat TP53 mutant MDS and AML patients are incredibly exciting, said trial principal investigator Asmita Mishra, M.D., of the H. Lee Moffitt Cancer Center and Research Institute.
  • Post-transplant maintenance therapy with eprenetapopt and azacitidine could, if approved, represent a new treatment paradigm that meaningfully improves outcomes for these patients with limited treatment options.
  • A Phase 1/2 clinical trial of eprenetapopt with venetoclax and azacitidine for the frontline treatment of TP53 mutant AML met the primary efficacy endpoint of complete remission.

NOXXON Pharma Enters Second Clinical Collaboration With MSD to Evaluate NOX-A12 in Combination With KEYTRUDA® (Pembrolizumab) in Upcoming Phase 2 Pancreatic Cancer Study

Retrieved on: 
Wednesday, July 21, 2021

This Phase 2 study will evaluate the safety and efficacy of NOX-A12, NOXXONs anti-CXCL12 agent, in combination with KEYTRUDA and two different chemotherapy regimens in patients with microsatellite stable pancreatic cancer.

Key Points: 
  • This Phase 2 study will evaluate the safety and efficacy of NOX-A12, NOXXONs anti-CXCL12 agent, in combination with KEYTRUDA and two different chemotherapy regimens in patients with microsatellite stable pancreatic cancer.
  • The vast majority of pancreatic cancer patients have microsatellite stable tumors which are resistant to checkpoint inhibitor monotherapy.
  • MSD will provide pembrolizumab and expert advice for the study protocol, while NOXXON will sponsor the trial that will be conducted in clinical centers in the US and Europe.
  • NOX-A12 is currently developed in second-line pancreatic cancer in combination with Keytruda and in glioblastoma in combination with radiotherapy.

RareStone Inc. Announces Submission of New Drug Application (NDA) of Wakix® (pitolisant) for narcolepsy with and without cataplexy in China

Retrieved on: 
Wednesday, July 21, 2021

Wakixwill be considered for approval in the treatment of narcolepsy with and without cataplexy in Greater China.

Key Points: 
  • Wakixwill be considered for approval in the treatment of narcolepsy with and without cataplexy in Greater China.
  • Through a partnership announced in Q4 2020 with Bioprojet, RareStone has exclusive rights for the commercialization and development of Wakixin China.
  • RareStone's lead product candidate, Wakix (pitolisant), is an investigational oral drug in development for the treatment of narcolepsy and obstructive sleep apnea in China.
  • Pitolisant has an orphan designation in the EU and the US for the treatment of narcolepsy.

bluebird bio Receives EC Approval for SKYSONA™ (elivaldogene autotemcel, Lenti-D™) Gene Therapy for Patients Less Than 18 Years of Age With Early Cerebral Adrenoleukodystrophy (CALD) Without Matched Sibling Donor

Retrieved on: 
Wednesday, July 21, 2021

bluebird bio, Inc. (Nasdaq: BLUE) today announced that the European Commission (EC) has granted marketing authorization of SKYSONA™ (elivaldogene autotemcel, Lenti-D™), a one-time gene therapy for the treatment of early cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA)-matched sibling hematopoietic (blood) stem cell (HSC) donor is not available. SKYSONA is the first one-time gene therapy approved in the European Union (EU) to treat CALD, a rare neurodegenerative disease that occurs in childhood and can rapidly lead to progressive, irreversible loss of neurologic function, and death.

Key Points: 
  • SKYSONA is a one-time gene therapy custom-designed to treat the underlying cause of the neurologic condition CALD.
  • SKYSONA uses ex vivo transduction with the Lenti-D lentiviral vector (LVV) to add functional copies of the ABCD1 gene into a patients own HSCs.
  • The addition of the functional ABCD1 gene allows patients to produce the ALD protein (ALDP), which is thought to facilitate the breakdown of VLCFAs.
  • The marketing authorization of SKYSONA is supported by efficacy and safety data from the Phase 2/3 Starbeam study (ALD-102).

FDA Grants Breakthrough Therapy Designation for Venclexta in Combination With Azacitidine for the Treatment of Patients With Myelodysplastic Syndromes

Retrieved on: 
Wednesday, July 21, 2021

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that Venclexta® (venetoclax) in combination with azacitidine has been granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with previously untreated intermediate, high- and very high-risk myelodysplastic syndromes (MDS) based on the revised International Prognostic Scoring System (IPSS-R). MDS are a rare group of blood cancers that gradually affect the ability of the bone marrow to produce normal blood cells. This can lead to weakness, frequent infections, anemia and debilitating fatigue that can profoundly affect a person’s quality of life. In some cases, MDS can also progress into acute myeloid leukemia (AML). Every year in the United States, approximately 10,000 people are diagnosed with MDS, and the median survival for those with higher-risk MDS is approximately 18 months.

Key Points: 
  • We are pleased that the FDA has granted Venclexta its sixth Breakthrough Therapy Designation in recognition of its potential to improve outcomes for people with MDS in combination with azacitidine.
  • The patients doctor will do blood tests to check for TLS when the patient first starts treatment and during treatment with Venclexta.
  • Patients should not receive a live vaccine before, during, or after treatment with Venclexta, until the patients doctor tells them it is okay.
  • The patients doctor will do blood tests to check their blood counts during treatment with Venclexta and may pause dosing.