New Hematology Drugs


new drugs in hematology

During the last two decades, we have witnessed the approval of numerous new hematology drugs and a complete transformation in how we approach blood cancers. A deeper understanding of the biology of specific hematologic malignancies has exposed numerous new therapeutic targets that were quickly followed by a plethora of novel therapeutic agents. The availability of novel drugs not only continues to improve patient outcomes but also generate numerous clinical questions. How to best combine or sequence new therapeutic agents? How to balance toxicity with the need for prolonged therapy? How cancer cells escape pharmacological suppression of a presumed vital pathway? How to make therapeutic innovations affordable to most patients? How to ensure barriers for clinical trial participation are removed so patients have early access to disease-modifying agents while answering the relevant clinic questions?

FDA Approved Drugs for Hematology

Drugs Approved in 2018

Andexxa (coagulation factor Xa (recombinant), inactivated-zhzo); Portola Pharmaceuticals; for the reversal of factor Xa inhibitors, Approved May 2018.

Adexxa (andexanet alfa) works by acting as a decoy for Factor Xa inhibitors in the blood, thereby preventing them from inhibiting the activity of native Factor Xa. As a result, the native Factor Xa is available to participate in the coagulation process and restore hemostasis (normal clotting).

Andexxa is specifically indicated for patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.

Andexxa is supplied as a lyophilized powder for solution for intravenous injection. Dose Andexxa based on the specific FXa inhibitor, the dose of FXa inhibitor, and time since the patient’s last dose of FXa inhibitor. Administer as an intravenous (IV) bolus, with a target rate of 30 mg/min, followed by continuous infusion for up to 120 minutes. There are two dosing regimens: Low Dose: Initial IV Bolus: 400 mg at a target rate of 30 mg/min. Follow-On IV Infusion: 4mg/min for up to 120 minutes. High Dose: Initial IV Bolus: 800 mg at a target rate of 30 mg/min. Follow-On IV Infusion: 8mg/min for up to 120 minutes. Read more

Factor Xa Inhibitor

Doptelet (avatrombopag); AkaRx; For the treatment of thrombocytopenia in adults with chronic liver disease scheduled to undergo a procedure, Approved May 2018.

Doptelet (avatrombopag) is a thrombopoietin receptor agonist.

Doptelet is specifically indicated for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure.

Doptelet is supplied as a tablet for oral administration. Begin Doptelet dosing 10-13 days prior to the scheduled procedure. The recommended daily dose of Doptelet is based on the patient’s platelet count prior to the scheduled procedure (see below). Patients should undergo their procedure 5 to 8 days after the last dose of Doptelet. Doptelet should be taken orally once daily for 5 consecutive days with food. In the case of a missed dose, patients should take the next dose of Doptelet as soon as they remember. Patients should not take two doses at one time to make up for a missed dose and should take the next dose at the usual time the next day; all five days of dosing should be completed.

Recommended Dose and Duration:

Platelet Count (x109/L): Less than 40 Once Daily Dose; 60 mg (3 tablets) for 5 days.

Platelet Count (x109/L): 40 to less than 50 Once-Daily Dose: 40 mg (2 tablets) for 5 days.

Read more

Doptelet

Lokelma (sodium zirconium cyclosilicate); AstraZeneca; For the treatment of hyperkalemia, Approved May 2018.

Lokelma (sodium zirconium cyclosilicate) is a highly-selective, oral potassium-removing agent.

Lokelma is specifically indicated for the treatment of hyperkalemia in adults.

Lokelma is supplied as a solution for oral administration. The recommended dose is 10 g administered three times a day for up to 48 hours. Administer Lokelma orally as a suspension in water. For continued treatment, the recommended dose is 10 g once daily. Monitor serum potassium and adjust the dose of Lokelma based on the serum potassium level and desired target range. During maintenance treatment, the dose may be up-titrated based on the serum potassium level at intervals of 1-week or longer and in increments of 5 g. The dose of Lokelma should be decreased or discontinued if the serum potassium is below the desired target range. The recommended maintenance dose range is from 5 g every other day to 15 g daily. Read more

sodium zirconium cyclosilicate

Palynziq (pegvaliase-pqpz); BioMarin; For the treatment of phenylketonuria, Approved May 2018.

Palynziq (pegvaliase-pqpz) is a phenylalanine-metabolizing enzyme.

Playnziq is specifically indicated to reduce blood phenylalanine concentrations in adult patients with phenylketonuria who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management. Read more

pegvaliase-pqpz

Tavalisse (fostamatinib disodium hexahydrate); Rigel Pharmaceuticals; For the treatment of chronic immune thrombocytopenia, Approved April 2018.

Tavalisse (fostamatinib disodium hexahydrate) is a tyrosine kinase inhibitor with demonstrated activity against spleen tyrosine kinase (SYK).

Tavalisse is specifically indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.

Tavalisse is supplied as a tablet for oral administration. Tavalisse should be initiated at a dose of 100 mg taken orally twice daily. After a month, if platelet count has not increased to at least 50 x 109/L, increase the Tavalisse dose to 150 mg twice daily. Use the lowest dose of Tavalisse to achieve and maintain a platelet count at least 50 x 109/L as necessary to reduce the risk of bleeding. Tavalisse may be taken with or without food. Read more

new drugs in hematology

Drugs Approved in 2017

Aliqopa (copanlisib); Bayer; For the treatment of follicular lymphoma, Approved September 2017.

Aliqopa (copanlisib) is a kinase inhibitor with activity predominantly against the PI3K-alpha and PI3K-delta isoforms expressed in malignant B cells.

Aliqopa is specifically indicated for the treatment of adult patients with relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies.

Aliqopa is supplied as a solution for intravenous infusion. The recommended dose is 60 mg administered as a 1-hour intravenous infusion on Days 1, 8, and 15 of a 28-day treatment cycle on an intermittent schedule (three weeks on and one week off). Continue treatment until disease progression or unacceptable toxicity. Read more

Copanlisib

Besponsa (inotuzumab ozogamicin); Pfizer; For the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia, Approved August 2017.

Besponsa (inotuzumab ozogamicin) is a CD22-directed antibody-drug conjugate (ADC).

Besponsa is specifically indicated for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.

Besponsa is supplied as a solution for intravenous injection. Read more

inotuzumab ozogamicin

Calquence (acalabrutinib); Acerta Pharmaceuticals; For the treatment of mantle cell lymphoma, Approved November 2017.

Calquence (acalabrutinib) is a small molecule Bruton tyrosine kinase inhibitor.

Calquence is specifically indicated for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.

Calquence is supplied as a capsule for oral administration. The recommended dose is 100 mg orally approximately every twelve hours. The capsule should be swallowed whole with water and with or without food. Read more

acalabrutinib

Endari (L-glutamine oral powder); Emmaus Life Sciences; For the treatment of sickle cell disease, Approved July 2017.

Endari (L-glutamine oral powder) reduces oxidant damage to red blood cells by improving the redox potential of nicotinamide adenine dinucleotide (NAD), a coenzyme that has been identified as the primary regulator of oxidation.

Endari is specifically indicated to reduce the severe complications of sickle cell disease (SCD) in adult and pediatric patients age 5 and older.

Endari is supplied as a powder for oral administration. Read more

L-glutamine oral powder

Giapreza (angiotensin II); La Jolla Pharmaceuticals; For the treatment of septic shock, Approved December 2017.

Giapreza (angiotensin II)  is a vasoconstrictor to increase blood pressure in adults with septic or other distributive shock.

Giaprezza is specifically indicated to increase blood pressure in adults with septic or other distributive shock.

Giapreza is supplied as a solution for intravenous administration. Dilute Giapreza in 0.9% sodium chloride prior to use.  See Full Prescribing Information for instructions on preparation and administration of injection. Giapreza must be administered as an intravenous infusion. Start Giapreza intravenously at 20 nanograms (ng)/kg/min. Titrate as frequently as every 5 minutes by increments of up to 15 ng/kg/min as needed. During the first 3 hours, the maximum dose should not exceed 80 ng/kg/min. The maintenance dose should not exceed 40 ng/kg/min. Read more

angiotensin II

Hemlibra (emicizumab-kxwh); Genentech; For the prevention or reduction of bleeding episodes in patients with hemophilia A, Approved November 2017.

Hemlibra (emicizumab-kxwh) is a bispecific factor IXa- and factor X-directed antibody.

Hemlibra is specifically indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors.

Hemlibra is supplied as a solution for subcutaneous injection. The recommended dose is 3 mg/kg by subcutaneous injection once weekly for the first 4 weeks, followed by 1.5 mg/kg once weekly. Read more

Emicizumab IDHIFA (enasidenib); Celgene; For the treatment of relapsed or refractory acute myeloid leukemia with IDH2 mutation, Approved August 2017.

IDHIFA (enasidenib) is a small molecule inhibitor of the isocitrate dehydrogenase 2 (IDH2) enzyme that works by blocking several enzymes that promote cell growth.

IDHIFA is specifically indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test.

IDHIFA is supplied as tablets for oral administration. The recommended starting dose of IDHIFA is 100 mg taken orally once daily with or without food until disease progression or unacceptable toxicity. For patients without disease progression or unacceptable toxicity, treat for a minimum of 6 months to allow time for clinical response.

Do not split or crush IDHIFA tablets.

Administer IDHIFA tablets orally about the same time each day. If a dose of IDHIFA is vomited, missed, or not taken at the usual time, administer the dose as soon as possible on the same day, and return to the normal schedule the following day. Read more

Enasidenib

Kymriah (tisagenlecleucel); Novartis; For the treatment of refractory B-cell precursor acute lymphoblastic leukemia, Approved August 2017.

Kymriah (tisagenlecleucel) is a CD19-directed genetically modified autologous T cell immunotherapy.

Kymriah is specifically indicated for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.

Kymriah is supplied as a suspension for intravenous infusion. Prior to infusion: premedicate with acetaminophen and an H1-antihistamine and confirm the availability of tocilizumab in the event of a CRS reaction. Dosing is based on the number of chimeric antigen receptor (CAR) positive viable T cells. For patients 50 kg or less, administer 0.2 to 5.0 x 10(6) CAR-positive viable T cells per kg body weight intravenously. For patients above 50 kg, administer 0.1 to 2.5  x 10(8) total CAR-positive viable T cells (non-weight based) intravenously. Read more

Tisagenlecleucel

Rebinyn (Coagulation Factor IX (Recombinant), GlycoPEGylated); Novo Nordisk; For the treatment of hemophilia B, Approved June 2017.

Rebinyn is a proprietary glycopegylated recombinant factor IX for patients with hemophilia B. Rebinyn is specifically indicated for use in adults and children with hemophilia B for on-demand treatment and control of bleeding episodes and for the perioperative management of bleeding.

Rebinyn is supplied as a lyophilized powder for solution for intravenous injection. The recommended dose for on-demand treatment and control of bleeding episodes is 40 IU/kg body weight for minor and moderate bleeds, and 80 IU/kg body weight for major bleeds. Additional doses of 40 IU/kg can be given. The recommended dose for perioperative management: Pre-operative dose of 40 IU/kg body weight for minor surgery, and 80 IU/kg body weight for major surgery. As clinically needed for the perioperative management of bleeding, repeated doses of 40 IU/kg (in 1-3 day intervals) within the first week after major surgery may be administered. Frequency may be extended to once weekly after the first week until bleeding stops and healing is achieved. Read more

glycopegylated recombinant factor IX

Rydapt (midostaurin); Novartis; For the treatment of FLT3 positive acute myeloid leukemia and mastocytosis , Approved April 2017.

Rydapt (midostaurin) is a multikinase inhibitor.

Rydapt is specifically indicated for the following:

  • in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation chemotherapy, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) who are FLT3 mutation positive, as detected by a FDA approved test
  • for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with associated hematological neoplasm (SM-AHN), or mast cell leukemia

Rydapt is supplied as a capsule for oral administration. Recommended Dosage in Acute Myeloid Leukemia: 50 mg orally twice daily with food on Days 8 to 21 of each cycle of induction with cytarabine and daunorubicin and on Days 8 to 21 of each cycle of consolidation with high-dose cytarabine. Recommended Dosage in ASM, SM-AHN, and MCL: 100 mg orally twice daily with food. Continue treatment until disease progression or unacceptable toxicity occurs.

Please see drug label for recommendations for dose modifications in patients with ASM, SM-AHN, and MCL. Monitor patients for toxicity at least weekly for the first 4 weeks, every other week for the next 8 weeks, and monthly thereafter while on treatment. Read more

Midostaurin

Vyxeos (daunorubicin and cytarabine); Jazz Pharma; For the treatment of newly-diagnosed therapy-related AML or AML with myelodysplasia-related changes, Approved August 2017.

Vyxeos is a liposomal combination of daunorubicin, an anthracycline topoisomerase inhibitor, and cytarabine, a nucleoside metabolic inhibitor.

Vyxeos is specifically indicated for the treatment of adults with newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

Vyxos is supplied as a liposome for intravenous infusion. The recommended dose schedule is as follows: Induction: Vyxeos (daunorubicin 44 mg/m2 and cytarabine 100 mg/m2) liposome via intravenous infusion over 90 minutes on days 1, 3, and 5 and on days 1 and 3 for subsequent cycles of induction, if needed. Consolidation: Vyxeos (daunorubicin 29 mg/m2 and cytarabine 65 mg/m2) liposome via intravenous infusion over 90 minutes on days 1 and 3. Read more

daunorubicin and cytarabine

Yescarta (axicabtagene ciloleucel); Kite Pharmaceuticals; For the treatment of relapsed or refractory large B-cell lymphomas, Approved October 2017.

Yescarta (axicabtagene ciloleucel) is a CD19-directed genetically modified autologous T cell immunotherapy.

Yescarta is specifically indicated for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.

Yescarta is supplied as a suspension for intravenous infusion. Each single infusion bag of Yescarta contains a suspension of chimeric antigen receptor (CAR)-positive T cells in approximately 68 mL. The target dose is 2 × 106 CAR-positive viable T cells per kg body weight, with a maximum of 2 × 108 CAR-positive viable T cells. Read more

axicabtagene ciloleucel

Drugs Approved in 2016

Afstyla (Antihemophilic Factor (Recombinant), Single Chain); CSL Behring; For the treatment of hemophillia A, Approved May 2016

Idelvion (Coagulation Factor IX (Recombinant), Albumin Fusion Protein); CSL Behring; For the treatment of hemophilia B, Approved March 2016

Kovaltry [Antihemophilic Factor (Recombinant)]; Bayer; For the treatment of hemophillia A, Approved March 2016

Opdivo (nivolumab); Bristol-Myers Squibb; For the treatment of classical Hodgkin lymphoma, Approved May 2016

Venclexta (venetoclax); AbbVie; For the treatment of chronic lymphocytic leukemia with 17p deletion, Approved April 2016

Drugs Approved in 2015

Darzalex (daratumumab); Janssen Biotech; For the treatment of multiple myeloma, Approved November 2015

Farydak (panobinostat); Novartis; For the treatment of multiple myeloma, Approved February 2015

Natpara (parathyroid hormone); NPS Pharmaceuticals; For the control hypocalcemia in patients with hypoparathyroidism, Approved January 2015

Nuwiq (recombinant Factor VIII); Octapharma; For the prophylaxis and treatment of hemophilia A, Approved September 2015

Praxbind (idarucizumab); Boehringer Ingelheim; For the reversal of the anticoagulant effects of dabigatran, Approved October 2015

Veltassa (patiromer); Relypsa; For the treatment of hyperkalemia, Approved October 2015

Drugs Approved in 2014

Alprolix [Coagulation Factor IX (Recombinant), Fc Fusion Protein]; Biogen Idec; For the treatment of hemophilia B, Approved March 2014

Beleodaq (belinostat); Spectrum Pharmaceuticals; For the treatment of relapsed or refractory peripheral T-cell lymphoma, Approved July 2014

Blincyto (blinatumomab); Amgen; For the treatment of Philadelphia chromosome-negative relapsed /refractory B cell precursor acute lymphoblastic leukemia, Approved December 2014

Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein] ; Biogen IDEC.; For the treatment of hemophillia A, Approved June 2014

Imbruvica (ibrutinib); Pharmacyclics; For the treatment of chronic lymphocytic leukemia and Waldenstrom macroglobulinemia, Approved February 2014

Obizur [Antihemophilic Factor (Recombinant), Porcine Sequence]; Baxter; For the treatment of acquired hemophilia A, Approved October 2014

Ruconest (C1 esterase inhibitor [recombinant]); Pharming Group; For the treatment of hereditary angioedema, Approved July 2014

Sylvant (siltuximab); Janssen Biotech; For the treatment of multicentric Castleman’s disease, Approved April 2014

Zontivity (vorapaxar); Merck; For the reduction of thrombotic cardiovascular events, Approved May 2014

Zydelig (idelalisib); Gilead; For the treatment of relapsed CLL, follicular B-cell NHL and small lymphocytic lymphoma, Approved July 2014

Drugs Approved in 2013

Gazyva (obinutuzumab); Genentech; For the treatment of previously untreated chronic lymphocytic leukemia, Approved October of 2013

Imbruvica (ibrutinib); Pharmacyclics; For the treatment of mantle cell lymphoma, Approved November of 2013

Injectafer (ferric carboxymaltose injection); Luitpold Pharmaceuticals; For the treatment of iron deficiency anemia, Approved July 2013

Kcentra (Prothrombin Complex Concentrate); CSL Behring; For the reversal of vitamin K antagonist therapy-induced coagulation factor deficiency, Approved May 2013

Nymalize (nimodipine); Arbor Pharmaceuticals; For the reduction of incidence and severity of ischemic deficits following subarachnoid hemorrhage, Approved May 2013

Pomalyst (pomalidomide); Celgene; For the treatment of relapsed and refractory multiple myeloma, Approved February 2013

Ravicti (glycerol phenylbutyrate); Hyperion Therapeutics; For the treatment of pediatrics and adults with urea cycle disorders, Approved February 2013

Revlimid (lenalidomide); Celgene; For the treatment of mantle cell lymphoma, Approved June 2013

Rixubis (Coagulation Factor IX (Recombinant)]; Baxter International; For the routine prophylaxis and control of hemophilia B, Approved June 2013

Tretten (Coagulation Factor XIII A-Subunit [Recombinant]); Novo Nordisk; For the treatment of congenital factor XIII (FXIII) A-subunit deficiency, Approved December of 2013

Valchlor (mechlorethamine) gel; Ceptaris Therapeutics; For the treatment of Stage IA/IB mycosisfungoides-type cutaneous T-cell lymphoma, Approved August 2013

Drugs Approved in 2012

Bosulif (bosutinib); Pfizer; For the treatment of Ph+ chronic myelogenous leukemia, Approved September 2012

Elelyso (taliglucerase alfa); Pfizer; For the treatment of Gaucher disease, Approved May 2012

Iclusig (ponatinib); Ariad Pharmaceuticals; For the treatment of chronic myeloid leukemia and Philadelphia chromosome positive acute lymphoblastic leukemia, Approved December 2012

Kyprolis (carfilzomib); Onyx Pharmaceuticals; For the treatment of multiple myeloma, Approved July 2012

Marqibo (vinCRIStine sulfate LIPOSOME injection); Talon Therapeutics; For the treatment of Ph- acute lymphoblastic leukemia, Approved August 2012

Neutroval (tbo-filgrastim); Teva Pharmaceutical; For the reduction in the duration of severe chemotherapy-induced neutropenia, Approved August 2012

Omontys (peginesatide); Affymax; For the treatment of anemia due to chronic kidney disease, Approved March 2012

Synribo (omacetaxine mepesuccinate); Teva Pharmaceutical; For the treatment of chronic or accelerated phase chronic myeloid leukemia, Approved October 2012

Drugs Approved in 2011

Adcetris (brentuximab vedotin); Seattle Genetics; For the treatment of Hodgkin lymphoma and anaplastic large cell lymphoma, Approved August 2011

Erwinaze (asparaginase Erwinia chrysanthemi); Eusa Pharma; For the treatment of acute lymphoblastic leukemia, Approved November of 2011

Ferriprox (deferiprone); Apotex; For the treatment of transfusional iron overload due to thalassemia, Approved October 2011

Jakafi (ruxolitinib); Incyte; For the treatment of myelofibrosis, Approved November 2011

Xarelto (rivaroxaban); Bayer; For the prophylaxis of deep vein thrombosis during knee or hip replacement surgery, Approved July 2011

Drugs Approved in 2010

Vpriv (velaglucerase alfa for injection); Shire Pharmaceuticals; For the treatment of type 1 Gaucher disease, Approved March 2010

References:

FDA Approved Drugs in Hematology | CenterWatch https://www.centerwatch.com/drug-information/fda-approved-drugs/therapeutic-area/6/hematology#.WzauJO7jvLY

New Drugs in Hematology http://www.mdpi.com/journal/pharmaceuticals/special_issues/Hematology

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