Hematological Disorders

FDA to Review Doptelet for Treatment of Chronic Immune Thrombocytopenia

By November 07, 2018

Doptelet is a thrombopoietin receptor agonist (TPO-RA) that was approved in May 2018 to treat thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a procedure.

FDA Approves Biosimilar Udenyca for Patients Receiving Myelosuppressive Chemotherapy

By November 05, 2018

Udenyca will be supplied as 6mg/0.6mL preservative-free solution in prefilled syringes for subcutaneous injection.

Jakafi Granted Priority Review for Acute Graft-Versus-Host Disease

By October 25, 2018

The sNDA included data from the REACH1 study (N=71) which evaluated ruxolitinib in combination with corticosteroids in patients with steroid-refractory acute GVHD.

BTK Inhibitor Designated Orphan Drug for Immune Thrombocytopenic Purpura

By October 19, 2018

PRN1008 is an oral, reversible covalent Bruton's tyrosine kinase (BTK) inhibitor; BTK is present in the signaling pathways of most types of white blood cells except for T cells and plasma cells.

Product Availability Update for Gammaplex Immune Globulin

By October 19, 2018

Gammaplex, an immune globulin, is indicated to treat chronic immune thrombocytopenic purpura (ITP) and primary humoral immunodeficiency.

FDA Approves New DNA-Based Test to Verify Blood Compatibility

October 15, 2018

If blood with poorly matched antibodies is transfused, the procedure is more likely to lead to red blood cell destruction and a transfusion reaction, the agency explained.

STRO-001 Gets Orphan Drug Designation for Mutiple Myeloma

By October 12, 2018

STRO-001 targets CD74, which is a protein highly expressed in B-cell malignancies like multiple myeloma.

High Flow Nasal Cannula Oxygen Tx May Benefit Elderly Patients in ICU

October 12, 2018

After high flow nasal cannula oxygen therapy, 15% of patients had to be intubated and mechanically ventilated, but the majority stepped down to regular oxygen therapy.

Anavip Now Available for the Management of Rattlesnake Envenomation

By October 09, 2018

Anavip contains venom-specific F(ab')2 fragments of immunoglobulin G (IgG) that bind and neutralize venom toxins, facilitating redistribution away from target tissues and elimination from the body.

Tegsedi Approved for Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis

By October 09, 2018

The approval was based on data from the Phase 3 NEURO-TTR study (N=173) which randomized patients with polyneuropathy caused by hATTR amyloidosis to Tegsedi or placebo.

FDA Approves Expanded Indication for Hemlibra in Hemophilia A

By October 04, 2018

The expanded approval was based on data from two Phase 3 clinical trials: HAVEN 3 and HAVEN 4.

Superwarfarin contaminants found in cannabinoid-associated coagulopathy outbreak

October 04, 2018

During one month at a single medical center in Illinois in 2018, 34 cases of synthetic cannabinoid associated coagulopathy were identified.

FDA Approves New Dosing Option for Kyprolis in Multiple Myeloma

By October 01, 2018

The approval was based on data from the Phase 3 A.R.R.O.W. trial, which demonstrated that Kyprolis 70mg/m2 once-weekly plus dexamethasone achieved superior progression-free survival (PFS) compared with Kyprolis 27mg/m2 twice-weekly with dexamethasone (median PFS: 11.2 months vs 7.6 months; hazard ratio [HR] 0.69; 95% CI, 0.54-0.88; one-sided P=.0014).

Oral Syk/JAK Inhibitor Gets Orphan Drug Designation for Peripheral T-Cell Lymphoma

By September 28, 2018

Preclinical data has suggested that Syk and JAK play important roles in PTCL tumor survival.

Hepcidin Mimetic Fast-Tracked for Beta-Thalassemia Treatment

By September 27, 2018

The Company is currently planning a Phase 2 trial of PTG-300 in beta-thalassemia patients which they expect to begin before the end of the year.

Labeling for Hereditary Factor X Deficiency Treatment Coagadex Updated

By September 26, 2018

In an open-label, non-randomized clinical trial, the use of Coagadex in routine prophylaxis of bleeding episodes was evaluated in 9 children <12 years of age.

New Risk Factors Identified for Varicose Vein Disease

September 25, 2018

Researchers applied machine learning to agnostically search for varicose vein risk factors in a cohort of 493,519 individuals in the UK Biobank.

FDA Requests Another Clinical Trial for Ruconest in HAE Prophylaxis

By September 21, 2018

The Company filed the sBLA last November following FDA feedback on two completed Phase 2 trials of Ruconest for prophylaxis of HAE attacks.

FDA Approves New Stent System to Treat Acute Coronary Artery Perforations

By September 17, 2018

The Agency reviewed real-world data from 80 patients who received the PK Papyrus Stents, finding that the stents were delivered successfully to the perforation site in 95% of patients with successful seals seen in 73 patients 91.3% of patients.

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.

Doptelet sNDA Submitted to FDA for Treatment of Immune Thrombocytopenia

By September 05, 2018

The sNDA includes safety and efficacy data from a Phase 3, randomized, placebo-controlled trial that met its primary efficacy endpoint of number of weeks with a platelet count ≥50x109/L in the absence of rescue therapy with high statistical significance.

Fostamatinib Seems Effective for Immune Thrombocytopenia

September 05, 2018

Forty-three percent of patients on fostamatinib and 14% on placebo achieved overall responses (defined as ≥1 platelet count ≥50,000/μL within the first 12 weeks on treatment).

Jivi With Step-Wise Dosing Approved for Management of Hemophilia A

By August 30, 2018

The approval was supported by data from the phase 2/3 international, open-label PROTECT VIII trial (N=126) that evaluated previously treated patients aged ≥12 years with severe hemophilia A.

Treatment Launched for Thrombocytopenia in Adults With Chronic Liver Disease

By August 30, 2018

Thrombocytopenia is a common complication of CLD, due to increased bleeding it can impact upon medical procedures. Mulpleta is supplied in 3mg strength tablets in blister packs containing 7 tablets.

Single-Administration Treatment for Hereditary Angioedema Gets Orphan Drug Status

By August 28, 2018

ADVM-053 is being developed as a single‑administration treatment that could potentially provide sustained levels of the C1 esterase inhibitor protein, which may prevent breakthrough angioedema attacks.

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.

First-of-its-Kind Therapy Approved to Prevent Hereditary Angioedema Attacks

By August 24, 2018

The approval was based on data from 4 clinical trials, including the 26-week Phase 3 HELP (Hereditary Angioedema Long-Term Prophylaxis) study that enrolled 125 patients with HAE aged ≥12 years.

Siklos Now Available for Pediatric Patients With Sickle Cell Anemia

By August 21, 2018

Hydroxyurea, an antimetabolite, is thought to cause an immediate inhibition of DNA synthesis by acting as a ribonucleotide reductase inhibitor, without interfering with the synthesis of ribonucleic acid or of protein.