Gralise (gabapentin; Depomed) is an extended-release formulation of gabapentin, an analog of the inhibitory neurotransmitter gamma-aminobutyric acid (GABA), and is used for treatment of restless leg syndrome and post-herpetic neuralgia (PHN).
Donaperminogene seltoplasmid (ViroMed), also referred to as VM202, is a DNA-based drug that is designed to produce two isoforms of HGF (hepatocyte growth factor), HGF728 and HGF723. When VM202 is delivered to the affected area by a single intramuscular injection, the drug enters a small portion of the surrounding muscle cells.
Cebranopadol is a novel small molecule analgesic, which was discovered by Grünenthal. Cebranopadol represents a novel, first-in-class potent analgesic that has a dual mechanism of action as a potent agonist of both the nociceptin/orphanin FQ peptide receptor (NOP) and opioid peptide receptor.
IMO-2125 is a synthetic phosphorothioate oligonucleotide-based agonist of toll-like receptor 9 (TLR9) designed to mimic the bacterial DNA that normally activates TLR9.
Seviprotimut-L (Polynoma/CK Life Sciences) is a polyvalent, shed-antigen vaccine in development for the adjuvant treatment of Stage IIb–III melanoma patients. Seviprotimut-L contains a combination of antigens from three proprietary melanoma cell lines, and is intended to stimulate the body’s immune system to fight cancer.
PDR001 (Novartis) is a fully humanized monoclonal antibody designed to inhibit the negative immunoregulatory receptor programmed death-1 (PD-1). PDR001 blocks the interaction between PD-1 and its ligands, programmed death-ligand 1 (PD-L1) and PD-L2; the signaling elicited by this interaction inhibits T-cell activation.
Daromun (Philogen) is a combination of Darleukin, a human vascular targeting monoclonal antibody (L19) fused to interleukin-2 (IL-2), and Fibromun, the L19 antibody linked to tumor necrosis factor (TNF). Both IL-2 and TNF are cytokines, key signaling molecules that are involved with the coordination of immunity.
Rebinyn is a glycoPEGylated rebombinant factor IX (rfIX) therapy developed by Novo Nordisk for the treatment of hemophilia B.
Hemlibra is an anti-factor IXa/X bispecific antibody, developed by Roche as a new treatment option for hemophilia A patients.
Valoctocogene roxaparvovec (BioMarin), also known as valrox, is an adeno-associated virus-mediated gene therapy that delivers human B domain-deleted coagulation factor VIII (fVIII) to patients with hemophilia A.
SPK-9001 is Spark Therapeutics’ leading candidate in development for hemophilia B.
Spark Therapeutics’ SPK-8011 is an adeno-associated virus-mediated gene therapy, aiming to restore normal clotting function to patients with hemophilia A.
Alnylam, in conjunction with its development partner Sanofi, is developing fitusiran for the treatment of hemophilia A and B.
AMT-061 is being developed by uniQure as a gene therapy for hemophilia B patients. The product uses an adeno-associated virus as a vector to deliver the highly active Padua factor IX (fIX) variant, which has eightfold to ninefold greater activity than normal, wild-type fIX.
Afstyla is a single-chain recombinant factor VIII (rfVIII) product developed by CSL Behring to have a longer duration of action than standard rfVIII. Endogenously produced fVIII consists of a light chain and a heavy chain fused via a metal ion bridge.
Nuwiq (Octapharma) is a B-domain deleted recombinant factor VIII (rfVIII) therapy developed for the treatment of adult and pediatric patients with hemophilia A.
Vonvendi (Shire) became the first recombinant von Willebrand factor (rVWF) therapy approved in the US in December 2015.
Lu AF35700 (Lundbeck) has a novel pharmacological profile as a dopamine D1 and D2 antagonist with a high occupancy of 5-HT2A and 5-HT6 serotonin receptors.
Nuplazid (Acadia Pharmaceuticals) contains pimavanserin, which is a selective serotonin inverse agonist preferentially targeting the serotonin 5-HT2A receptors.
MIN-101 (Minerva Neurosciences/Mitsubishi Tanabe) is a novel antagonist of the sigma-2 and serotonin 5-HT2A receptors.
Lonasen (Sumitomo Dainippon/Nitto Denko) contains blonanserin, a dopamine D2 and serotonin 5-HT2 receptor antagonist.
Trelegy Ellipta ([fluticasone furoate + vilanterol + umeclidinium bromide]; GlaxoSmithKline) is a fixed triple combination inhaled corticosteroid/long-acting beta 2 agonist/long-acting muscarinic antagonist (ICS/LABA/LAMA) therapy.
Fasenra (benralizumab; AstraZeneca/Kyowa Hakko Kirin) is a humanized monoclonal antibody which binds to the alpha chain of the interleukin (IL)-5 receptor on eosinophils and therefore depletes them.
Dupixent (dupilumab; Regeneron/Sanofi) is an injectable monoclonal antibody directed against the interleukin (IL)-4 receptor alpha subunit, which blocks signaling from both IL-4 and IL-13.
Bevyxxa (betrixaban; Portola Pharmaceuticals) is approved in the US for the prophylaxis of VTE in adult patients hospitalized for an acute medical illness.
Zoladex (goserelin; AstraZeneca/TerSera Therapeutics) is a gonadotropin-releasing hormone (GnRH) receptor agonist which acts on the pituitary gland to stimulate luteinizing hormone (LH) release, resulting in a temporary surge in serum testosterone levels known as “flare.”
Trelstar (triptorelin; Allergan) is a gonadotropin-releasing hormone (GnRH) receptor agonist which acts on the pituitary gland to stimulate luteinizing hormone (LH) release, resulting in a temporary surge in serum testosterone levels known as “flare.”
Firmagon (degarelix; Ferring/Astellas) is a gonadotropin-releasing hormone (GnRH) receptor antagonist that directly binds to GnRH receptors in the anterior pituitary gland, rapidly reducing the circulating gonadotropins luteinizing hormone (LH) and follicle-stimulating hormone (FSH), and leading to the rapid suppression of testosterone in men.
Eligard (leuprolide; Astellas/Tolmar Pharmaceuticals) is a gonadotropin-releasing hormone (GnRH) receptor agonist which acts on the pituitary gland to stimulate luteinizing hormone (LH) release, resulting in a temporary surge in serum testosterone levels known as “flare.”
Yonsa (Churchill Pharmaceuticals) is an oral, ultramicrosize tablet formulation of abiraterone acetate that inhibits 17 alpha-hydroxylase (CYP17). The CYP17 family of enzymes catalyze the hydroxylation of intermediates involved in testosterone synthesis, a common driver of prostate cancer genesis and recurrence.
Relugolix (Myovant Sciences/Takeda) is a gonadotropin-releasing hormone (GnRH) receptor antagonist that directly inhibits GnRH receptors, rapidly reducing the circulating gonadotropin’s luteinizing hormone (LH) and follicle-stimulating hormone (FSH) and leading to the suppression of testosterone in men.
Ipatasertib (Roche) is a highly selective, ATP-competitive Akt inhibitor that preferentially targets active, phosphorylated Akt.
Apalutamide (Johnson & Johnson) is an orally available androgen signaling inhibitor. The drug exhibits high binding affinity to the ligand-binding domain of androgen receptor (AR) and subsequently inhibits its nuclear import, as well as its ability to bind to DNA. Apalutamide has a similar mechanism of action to Xtandi (enzalutamide; Pfizer/Astellas).
Darolutamide is a non-steroidal oral androgen receptor (AR)antagonist in development by Bayer for the treatment of prostate cancer. Darolutamide potently inhibits the binding of androgens to ARs, blocking nuclear translocation of ARs and AR-mediated gene expression. The AR signaling pathway is the primary pathway that drives prostate cancer growth.
Dexferrum (iron dextran; Luitpold Pharmaceuticals) is an intravenous (IV) iron maintenance treatment used to help elevate hemoglobin levels in patients with anemia in chronic kidney disease (CKD).
Feraheme (ferumoxytol; AMAG Pharmaceuticals) is an intravenous (IV) iron maintenance treatment used to help elevate hemoglobin levels in patients with anemia in chronic kidney disease (CKD).
Injectafer (ferric carboxymaltose; Vifor/Luitpold Pharmaceuticals/Zeria) is an iron maintenance treatment used to help elevate hemoglobin levels in patients with anemia in chronic kidney disease (CKD). It is administered intravenously and is dissociated into iron and sucrose once in circulation.
NeoRecormon (epoetin beta; Roche/Chugai) is an erythropoiesis-stimulating agent (ESA) administered by intravenous (IV) or subcutaneous (SC) injection, and is indicated for the treatment of anemia in chronic kidney disease (CKD).
Dengvaxia (tetravalent live-attenuated chimeric vaccine; Sanofi Pasteur) is a tetravalent vaccine comprised of four monovalent, chimeric live-attenuated vaccines (LAVs).
Omecamtiv mecarbil is a novel cardiac myosin activator that increases contractility in heart muscle through selectively activating the enzyme myosin ATPase.
Vericiguat is a first-in-class soluble guanylate cyclase (sGC) stimulator that acts to increase levels of cyclic guanosine monophosphate (cGMP) via the nitric oxide-sGC-cGMP pathway.
Ofatumumab (Genmab/Novartis) is a fully human immunoglobulin G1 and high-affinity antibody that targets a novel epitope on cluster of differentiation (CD)20 on the B-cell membrane. Ofatumumab binds effectively to both the small and large extracellular loops on CD20 and releases very slowly over time.
ALKS 8700 is an oral monomethyl fumarate molecule being developed for the treatment of relapsing multiple sclerosis.
Ocrevus is a second-generation cluster of differentiation (CD)20 antibody. Similar to Roche’s blockbuster cancer drug Rituxan (rituximab), Ocrevus specifically targets CD20+ B cells, thus depleting lymphocytes that produce the autoimmune response.
Mavenclad contains cladribine, a small molecule that suppresses the immune system by selectively targeting lymphocytes, which are cells that seem to be major players in MS pathological mechanisms.
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