Hematological Disorders

CPI-613 Gets Orphan Drug Designation for Peripheral T-Cell Lymphoma

By August 17, 2018

This first-in-class drug was previously granted Orphan Drug designation for the treatment of pancreatic cancer, acute myeloid leukemia, Burkitt lymphoma, and myelodysplastic syndromes.

Elzonris Granted Priority Review for Blastic Plasmacytoid Dendritic Cell Neoplasm

By August 14, 2018

The Company announced results from a Phase 2 trial involving 45 patients with BPDCN earlier this year.

Supplemental BLA Submitted for Darzalex Split Dosing Regimen

By August 09, 2018

The proposed dosing regimen would provide the option of splitting the first infusion of Darzalex over 2 consecutive days.

Antidepressants and Bleeding Risk: What's the Link?

By August 08, 2018

Results showed an increased risk of bleeding with SRIs by 1.16-2.36 times with an even higher 3.17- to 10.9-fold risk with concomitant NSAIDs.

FDA Fast-Tracks Plasma Kallikrein Inhibitor for Angioedema Attacks in HAE

By August 08, 2018

The Company is currently conducting a Phase 3 trial (APeX-2) and a long-term safety study (APeX-S) evaluating 2 doses of the treatment.

Granix Indication Expanded to Include Pediatric Patients

By August 07, 2018

The pediatric approval was supported by evidence from studies in adults with additional safety and pharmacokinetics data from a single-arm trial of 50 pediatric patients with solid tumors treated with Granix for chemotherapy-induced neutropenia.

Thromboembolic Disorder Treatments: Without DVT/PE

August 06, 2018

A comparative medication chart of thromboembolic disorder treatments in patients without DVT/PE.

Thromboembolic Disorder Treatments: DVT/PE

August 06, 2018

List of medications indicated for the prophylaxis and/or treatment of deep vein thrombosis (DVT) / pulmonary embolism (PE) with usual adult dosage.

Treatment for FLT3-ITD Acute Myeloid Leukemia Gets Breakthrough Designation

By August 02, 2018

The designation was based on results from the Phase 3 QuANTUM-R trial, which included 367 participants who were randomized to receive quizartinib or salvage chemotherapy (SC).

Anti-CD47 Antibody Granted Orphan Drug Designation for Multiple Myeloma

By August 01, 2018

SRF231 is a fully human monoclonal antibody therapeutic targeting CD47, a protein overexpressed on many cancer cells which prevents them from being engulfed and eliminated by macrophage mediated phagocytosis.

Mulpleta Approved for Thrombocytopenia in Patients With Chronic Liver Disease

By July 31, 2018

The approval was based on data from 2 randomized, double-blind, placebo-controlled trials (L-PLUS 1 [N=97] and L-PLUS 2 [N=215]) involving patients with chronic liver disease who were undergoing an invasive procedure and had a platelet count <50 x 109/L.

Iron Therapies Compared in Patients With GI-Related Iron Deficiency Anemia

By July 30, 2018

The analysis also showed that the more severe the anemia was at baseline, the greater the response was to treatment with regard to hemoglobin changes.

Plasma Kallikrein Inhibitor Cuts Rate of Hereditary Angioedema Attacks

July 26, 2018

The most commonly reported adverse events were gastrointestinal adverse events, mainly grade 1, especially in the 2 highest BCX7353-dose groups.

Biosimilar Nivestym Gets FDA Approval

By July 23, 2018

The approval was based on data demonstrating that the biosimilar product and the reference product are highly similar, and that there are no clinically meaningful differences between the 2 agents.

Tibsovo Approved for Relapsed/Refractory AML With IDH1 Mutation

By July 20, 2018

The approval of Tibsovo was based on an open-label, single-arm, multicenter clinical trial (Study AG120-C-001) involving 174 patients with relapsed or refractory AML with an IDH1 miutation.

Galinpepimut-S Fast-Tracked for Multiple Myeloma Treatment

By July 20, 2018

As WT1 is present in an array of tumor types, the Company believes GPS has the potential to be a broad immunotherapy.

Bone Marrow Transplant Alternative Granted Orphan Drug Designation

By July 17, 2018

It is also the first bone marrow transplant alternative to be granted Breakthrough Therapy status by the FDA.

Dilanubicel Gets Orphan Drug Status for Reduction of Morbidity/Mortality Associated With HSCT

By July 17, 2018

Dilanubicel is a universal donor, off-the-shelf-, ex vivo expanded hematopoietic stem and progenitor cell investigational product that provides rapid, transient hematopoiesis while also inducing long-term immunologic benefits.

ESA Biosimilar Effective for Anemia in Hemodialysis Patients

July 13, 2018

Researchers found "no clinically meaningful differences" in efficacy and safety between the biosimilar epoetin alfa-epbx and epoetin alfa.

FDA Drops Zika Testing for Blood Donors

July 10, 2018

"Now, given the significant decrease in cases of Zika virus infection in the United States and its territories, we are moving away from testing each individual donation to testing pooled donations."

Red Cross Issues Nationwide Call for Blood Donations

July 10, 2018

"Blood donations are currently being distributed to hospitals faster than donations are coming in - we need both new and current blood donors to make an appointment as soon as possible to help patients battling illness and injury," said Chris Hrouda, president of Red Cross Blood Services.

Glasdegib NDA Granted Priority Review for Acute Myeloid Leukemia

By June 28, 2018

Glasdegib, an investigational oral smoothened (SMO) inhibitor, is a once-daily therapy that is thought to work by disrupting the Hedgehog pathway.

Most Bleeding Events in Non-CVD Patients Are GI-Related

June 28, 2018

The risk of a nonfatal GI bleeding event was 2.19 per 1,000 person-years in the baseline cohort, 1.77 in the non-high-risk cohort, and 1.61 in the nonmedication cohort.

Clotting Time in Transfemoral PCI Linked to Bleeding Risk

June 27, 2018

Researchers related maximal ACT to the risk of major bleeding in 14,634 patients undergoing TR or TF PCI with unfractionated heparin monotherapy.

Combination Therapy for Waldenström's Macroglobulinemia Gets Priority Review

By June 26, 2018

The sNDA is supported by data from the Phase 3 iNNOVATE (PCYC-1127) trial which assessed ibrutinib in combination with rituximab vs rituximab alone in 150 patients with previously untreated and relapsed/refractory WM.

FDA to Review Afamelanotide NDA for Erythropoietic Protoporphyria

By June 25, 2018

Scenesse (Clinuvel Pharmaceuticals) is a bimonthly, subcutaneous dose of afamelanotide 16mg.

Cinryze Approved for Pediatric Patients With Hereditary Angioedema

By June 22, 2018

The approval was based on data from a randomized, single-blind, multicenter, dose-ranging, crossover study which evaluated the safety and efficacy of Cinryze in 12 pediatric patients (7 to 11 years old).

Keytruda Approved for Primary Mediastinal Large B-Cell Lymphoma

By June 14, 2018

The approval was based on data from the KEYNOTE-170 study, a multicenter, open-label, single-arm trial that included 53 patients with relapsed or refractory PMBCL.

Kyprolis Labeling Updated With Overall Survival Data From Multiple Myeloma Trial

By June 11, 2018

ASPIRE (CArfilzomib, Lenalidomide, and DexamethaSone versus Lenalidomide and Dexamethasone for the treatment of PatIents with Relapsed Multiple MyEloma) was a Phase 3 trial that evaluated the triplet regimen Kyprolis, lenalidomide, and dexamethasone (KRd) vs lenalidomide and dexamethasone (Rd) alone, in patients with relapsed or refractory multiple myeloma who received 1-3 prior regimens.

Combo Tx Approved for Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma

By June 11, 2018

The new approval was based results from the Phase 3 MURANO trial which included 389 patients with CLL who had received ≥1 prior line of therapy.