Miscellaneous Hematological Agents

First-in-Class Treatment Approved for Hairy Cell Leukemia

By September 14, 2018

The approval of Lumoxiti was based on data from a Phase 3 single-arm, open-label trial conducted in patients with histologically confirmed HCL or HCL variant with a need for therapy based on presence of cytopenias or splenomegaly and who had received prior treatment with at least 2 systemic therapies, including a purine nucleoside analog (N=80).

Venclexta Labeling Updated With Minimal Residual Disease Negativity Data

By September 11, 2018

MRD-negativity is defined as <1 CLL cell per 10,000 lymphocytes detected in the blood or bone marrow using sensitive analytical models.

Imbruvica Plus Rituximab Approved for Waldenström's Macroglobulinemia

By August 27, 2018

This expanded approval was based on results from the iNNOVATE study, a double-blind, placebo-controlled trial evaluating Imbruvica in combination with rituximab vs placebo + rituximab in 150 patients with either relapsed/refractory disease or previously untreated Waldenström's macroglobulinemia.

AML Treatment for Patients Over 60 Gets Fast Track Designation

By August 27, 2018

CX-01 is designed to block the activity of chemokines that support the resistance of blood cancers to treatment and that contribute to the delay of bone marrow recovery after chemotherapy.

Accuracy of Hypoxia-Based Baby Monitors Is Concerning

August 22, 2018

"As more neonate and infant vital sign monitors emerge in this largely unregulated market, physicians and parents should exercise caution incorporating data from these monitors into medical decisions."

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.

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.

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.

<small>L</small>-Glutamine Reduces Number of Pain Crises in Sickle Cell Disease

July 19, 2018

The researchers found that there were significantly fewer pain crises in the L-glutamine group than in the placebo group, with a median of 3.0 and 4.0, respectively.

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.

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.

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.

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.

Oral Propranolol Seems Safe for Infantile Hemangioma

By May 31, 2018

"In this study on a large continuous nationwide claims database, we confirm the safety profile of oral propranolol in healthy children to be good," the authors write.

CDC Warns of Serious Coagulopathy Outbreak Linked to Synthetic Cannabinoids

By May 29, 2018

Since brodifacoum - a long-acting vitamin K-dependent antagonist, used as a rodenticide - was detected in drug and biologic samples from patients using synthetic cannabinoids, there have been 202 reported cases of coagulopathy in 9 states (mostly Illinois; N=164)

FDA Committees Vote Against Drug Tx for Severe Neonatal Jaundice

By May 04, 2018

Stannsoporfin, a heme oxygenase inhibitor, works by inhibiting bilirubin production at its source.

Pharmacologic Tx for Severe Neonatal Jaundice to Be Reviewed by FDA

By February 23, 2018

The FDA has set an action date of August 22, 2018 to respond to the NDA

Treating Endothelial Function May Target Root Disease in Marfan Syndrome

February 13, 2018

The researchers found that the MFS/ATR1a-null mice showed unabated aortic root enlargement and remained responsive to losartan, despite being hypotensive and resistant to AngII vasopressor effects.

Antibiotic Prophylaxis Rates Low for Pediatric Sickle Cell Patients

February 06, 2018

Data were included for 2,821 children with 5,014 person-years. The researchers found that 18 percent of children received ≥300 days of antibiotics.

Results Released in Multi-Year IV Bevacizumab Study for HHT-Related Bleeding

February 05, 2018

After completion of the initial bevacizumab treatment cycle, there was a significant reduction in epistaxis severity scores and RBC transfusion requirements.