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.
Udenyca will be supplied as 6mg/0.6mL preservative-free solution in prefilled syringes for subcutaneous injection.
The sNDA included data from the REACH1 study (N=71) which evaluated ruxolitinib in combination with corticosteroids in patients with steroid-refractory acute GVHD.
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.
Gammaplex, an immune globulin, is indicated to treat chronic immune thrombocytopenic purpura (ITP) and primary humoral immunodeficiency.
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.
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 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.
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.
During one month at a single medical center in Illinois in 2018, 34 cases of synthetic cannabinoid associated coagulopathy were identified.
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).
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.
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.
Researchers applied machine learning to agnostically search for varicose vein risk factors in a cohort of 493,519 individuals in the UK Biobank.
The Company filed the sBLA last November following FDA feedback on two completed Phase 2 trials of Ruconest for prophylaxis of HAE attacks.
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.
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).
MRD-negativity is defined as <1 CLL cell per 10,000 lymphocytes detected in the blood or bone marrow using sensitive analytical models.
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.
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).
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.
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.
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.
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.
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.
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.
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.