s p e c i a l t y d r u g n e w s Positively affecting the lives of our members each and every day Efalizumab (Raptiva ) Withdrawn from US Market On April 9, 2009 Genentech, Inc. announced that it is undergoing a voluntary, phased withdrawal in US markets of Raptiva, due to the potential risk to patient of developing progressive multifocal leukoencephalopathy (PML). Efalizumab is used for treatment of chronic moderate or severe plaque psoriasis. PML is a rare and usually fatal viral disease characterized by progressive damage or inflammation to the white ma er of the brain at multiple locations. According to the FDA the risk that an individual patient taking efalizumab will develop PML is rare and is generally associated with long-term use. PML generally occurs in patients with immune systems that are severely weakened and o en leads to an irreversible decline in neurologic function and death. Efalizumab will no longer be available in the United States a er June 8, 2009, and healthcare providers are being asked not to initiate treatment in new patients. Physicians are also being asked to closely monitor patients who have been on the therapy for any sign or symptom that is suggestive of PML and for infections or other adverse events a er stopping treatment. FDA Grants Fast-Track Status for Multiple Sclerosis Drug Teva Pharmaceuticals has announced that the FDA has granted fast-track review status of oral laquinimod for treatment of multiple sclerosis. This designation should help expedite its future review by the FDA a er results of the current clinical trials are reviewed. MS occurs when the immune system mistakenly a acks the myelin sheath on nerve fibers, and also damages other brain and spinal cord tissues. Laquinimod is believed to affect this immune a ack. Laquinimod reduced disease activity by 40.4% compared to inactive placebo in earlier phase 2 studies. Volume 19 April 2009 In this issue: Efalizumab (Raptiva) Withdrawn from US Market FDA Grants Fast- Track Status for Multiple Sclerosis Drug Benchmarking Survey Shows Treating Patients in the Office with Specialty Drugs is Critical to Rheumatologists Revenue Streams 1st Quarter 2009 Speciatly Drug Pipeline Specialty Drug Listing Currently, the Allegro study, a phase 3 study of laquinimod versus inactive placebo in 1000 participants with relapsing-remi ing MS, is underway. A second phase 3 study, the Bravo study, of laquinimod in comparison to inactive placebo or Avonex is underway in 1200 participants.
Benchmarking Survey Shows Treating Patients in the Office with Specialty Drugs is Critical to Rheumatologists Revenue Streams A recent report from Onmark, a leading national group purchasing organization for community based-medical practices and a McKesson Specialty Care Solutions company, shows that rheumatologists rely on specialty drugs like Remicade for revenue streams. In a survey that measures operational and financial benchmarks to help practices improve business operations, efficiency and quality of care the following data was collected in 2007: General practice information Accounts receivable and general revenue data Operating costs Ancillary services Compensation The survey revealed that the sale and administration of drugs to patients represents 74% of the revenue of the practices surveyed. The total cost of drugs for in-office administration to patients accounts for an average of 62% of all practice expenses. The total average drug costs per practice is about $4,875,796, subtracting all rebates and excluding medications sold to patients for home use. The most commonly coded procedures from rheumatology offices are related to drug administration. *Source: Business Wire Drug Name RiaSTAP Atryn Promacta fludarabine Treanda Degarelix Mozobil SPECIALTY PIPELINE UPDATE Indication for Use Used in treating acute bleeding episodes in patients with congenital fibrinogen deficiency Prevention of thromboembolic events thrombocytopenia in patients with chronic ITP adults with relapsed or refractory B-cell CLL B-cell non-hodgkin's lymphoma advanced prostate cancer Used to mobilize stem cells for collection and autologous transplants in patients with NHL and mutiple myeloma Route of Approval Date Contact: For more information regarding LDI s Specialty Newsle er, please contact us: (314) 652-2121 x189 (866) 516-2121 x189 VBohn@LDIRx.com IV injection 1/16/09 First approved treatment for this indication IV injection 2/6/09 First approved biological prodcut produced by genetically engineered animals 11/20/08 First oral thrombopoietin receptor agonist approved for adults with chronic ITP 12/18/08 10/31/08 Previously approved for treatment of CLL 12/24/08 12/15/08 Potential to decrease number of apheresis days required to collect cells for transplant
PIPELINE MEDICATIONS IN PHASE III TRIALS Drug Name Indication for Use Route of Kiacta Hematide Bosatria amyloid A amyloidosis anemia in patients with chronic renal failure hypereosinophilic syndrome Injection Administered once every four weeks in clinical trials Denufosol cystic fibrosis Inhalation Orphan drug with fast track status. Primary endpoints achieved in first phase III trial in June 2008 prgcd (plant cell expressed recombinant glucocerebrosidase Gaucher disease Enrollment complete for phase III trial Velaglucerase alfa Albuferon Boceprevir Berinert P DX-88 (ecallantide) Rhucin Vicriviroc Tesamorelin Corifollitropin alfa Golimumab Ustekinumab Canakinumab type 1 Gaucher disease In combination with ribavirin for treatment of chronic hepatitis C In combination with Peg-Intron and Rebetol for treatment of hepatitis C acute a acks in patients with hereditary angioedema (HAE) moderate to severe acute HAE a acks acute a acks in patients with HAE HIV in combination with other antiretroviral agents HIV-associated lipodystrophy Worldwide enrollment for phase III trial completed Injection Phase III data expected by spring 2009 Enrollment complete for phase III trial with fast track status Two large phase III trials Infertility Primary endpoints met in phase III trial RA, psoriatic arthritis and ankylosing spondylitis Treatment in adults with chronic moderate to severe plaque psoriasis cryopyrin-associated syndromes
PIPELINE MEDICATIONS IN PHASE III TRIALS, continued Drug Name Indication for Use Route of Actemra Pegloticase Fampridine-SR Dirucotide Fingolimod Teriflunomide Laquinimod Pasireotide Zactima Opaxio Pixantrone Used in reducing signs and symptoms in adults with moderate to severe RA gout in patients for whom conventional treatment is contraindicted or ineffective Use to treat walking ability in patients with MS secondary-progressive MS relapsing-remitting MS relapsing forms of MS relapsing-remitting MS Cushing's disease and acromegaly Second line treatment of nonsmall cell lung cancer advanced nonsmall cell lung cancer in women and for maintenance treatment of ovarian cancer relapsed non- Hodgkin's lymphoma Lestaurtinib acute myeloid leukemia Provenge metastatic hormone-refractory prostate cancer Ceplene In conjunction with IL-2 as a remission maintenance treatment of AML Arzerra refractory CLL Genasense relapsed or refactory CLL Virulizin First-line treatment of pancreatic Intramuscular cancer in combination injection with Gemzar FDA has requested additional information, including a proposal for a Risk Evaluation and Mitigation Strategy FDA granted priority review status in December 2008 FDA granted fast track status Also being studied in combination with interferon-beta and Copaxone Phase III trial enrollment complete November 2008 FDA granted fast track status Designed to reduce the potential for heart damage compared to current anthracyclines FDA final analysis expected mid-2009 with fast track status
PIPELINE MEDICATIONS IN PHASE III TRIALS, continued Drug Name Indication for Use Route of Phenoxodiol hormonerefactory IV injection/oral FDA granted fast track status prostate cancer in Taxotere nonresponders and recurrent chemo resistant late-stage ovarian cancer Deforolimus metastatic sarcoma Afinitor advanced renal FDA granted priority review status cell carcinoma and neuroendocrine tumors Trabectedin In combination with Doxil for treatment of relapsed ovarian cancer Larotaxel Second-line treatment of pancreatic cancer Denosumab postmenopausal osteoporosis and cancer-related bone loss Thelin pulmonary arterial hypertension. Phase III study initiated in November 2008 Pirfenidone idiopathic pulmonary fibrosis Numax RSV IM injection Expected to be more potent than Synagis. FDA has requested more information. Certican Prevention of solid organ rejection in combination with Neoral Clinical trials ongoing NEW DOSAGE FORMS IN THE PIPELINE Drug Name Current Indication Investigational Indication Xolair Cinryze moderate to severe persistent allergic asthma (age 12 and above) Routine prophylaxis against angioedema a acks in patients with HAE children 6 years and above with moderate to severe persistent allergic asthma acute angioedema a acks in patients with HAE Route of FDA granted priority review status in February 2009
NEW DOSAGE FORMS IN THE PIPELINE, continued Drug Name Current Indication Investigational Indication Isentress H.P. Acthar Gel Orencia Rituxan In combination with other antiretroviral agents for treatment-experienced HIV patients who have evidence of viral replication and HIV strains resistant to multiple antiretroviral agents Multiple indications, including the diagnostic testing of adrenocortical function and the treatment of MS exacerbations moderately to severely active RA in adults and moderately to severe active polyarticular juvenile idiopathic arthritis in pediatric patients 6 years of age and older non-hodgkin's lymphoma and treatment of moderately to severely active RA in patients who have had an inadequate response to one or more TNF inhibitors In combination with other antiretroviral agents for patients with treatment-naïve HIV For treatment of infantile spasms patients with early RA moderately to severely active RA in patients who have had an inadequate response to prior treatment with a diseases modifying anti-rheumatic drug Cimzia Crohn's disease moderate to severe or active RA Zevalin relapsed or refractory, low-grade or follicular B-cell NHL Consolidation therapy for patients with follicular B- cell NHL who achieve a response to firstline therapy Route of IM or IV injection FDA response to supplemental new expected July 2009 resubmitted December 2008 filed in the fourth quarter 2008 filed October 2008 FDA has requested a new safety update with all clinical data FDA granted priority review status December 2008
NEW DOSAGE FORMS IN THE PIPELINE, continued Drug Name Current Indication Investigational Indication Avastin Clolar Tasigna Sutent Forteo Reclast breast cancer, colorectal cancer and nonsmall cell lung cancer pediatric patients (1 to 21 years old) with relapsed or refractory acute lymphoblastic leukemia after at least two prior regimens chronic and accelerated phase Philadelphia chromosome positive chronic myelogenous leukemia GIST and advanced RCC men and postmenopausal women with osteoporosis who are at high risk for fracture Paget's disease and postmenopausal osteoporosis First line treatment of renal cell carcinoma (in combination with interferon alfa-2a) and for treatment of relapsed glioblastoma multiforme adult patients with AML gastrointestinal stromal tumor (GIST) in patients who have failed both Gleevec and Sutent colorectal cancer, metastatic breast cancer and non-small cell lung cancer glucocorticoidinduced osteoporosis glucocorticoidinduced osteoporosis Route of Responses to supplemental new drug applications expected in May 2009 for glioblastoma and August 2009 for RCC drug filing expected in 2009 Phase III trials ongoing filed 2008
NEW DOSAGE FORMS IN THE PIPELINE Drug Name Indication Current Route of Investigational Route of C2L (octreotide) Cayston TBM 100 (tobramycin) Mylinax Trelstar Viveta acromegaly patients with cystic fibrosis who have pulmonary Pseudomonas aeruginosa patients with cystic fibrosis who have pulmonary Pseudomonas aeruginosa relapsing forms of MS For palliative treatment of advanced prostate cancer pulmonary arterial hypertension IM injection IM injection Prolonged release formulation designed to be dosed less frequently than the long-acting formulation (Sandostatin LAR) IV injection Inhalation. Available through expanded access program. Solution for inhalation Powder for inhalation Expected to provide rapid convenient administration of tobramycin. with fast track status IM Injection IM Injection This formulation is a sustainedrelease formulation designed to be administered every six months. SC or IV Inhalation Studied in combination with Tracleer infusion or Revatio
Specialty drug listing AIDS/HIV Daunoxome Doxil Intron A Roferon-A Serostim Taxol Fuzeon Alpha1 - Proteinase Inhibitor Deficiency Aralast Prolastin Zemaira Antihemophilic Agents Antihemophilic Factor Advate Alphanate Bioclate Helixate FS Hemofil M Humate P Hyate C Koate DVI Kogenate FS Monarc M Monoclate P Recombinate ReFacto Anti-nausea Aloxi Anzemet Emend Kytril Zofran Asthma Xolair Cancer/Related Adriamycin Adrucil Alkeran Aredia Avastin BiCNU Blenoxane Busulfex Campath Camptosar Cerubidine Cosmegen Cytarabine Cytoxan Depocyt Doxil DTIC-Dome Eligard Ellence Erbitux Ethyol Faslodex Fludara Gemzar Gleevec Herceptin Hycamtin Idamycin IFEX Intron A Leucovorin Leukine Leustatin Lupron Depot Lupron Depot-Ped Mesnex Mustargen Mutamycin Mylotarg Navelbine Nexavar Nipent Novantrone Oncaspar Ontak Paraplatin Platinol AQ Proleukin Rituxan Roferon-A Sutent Tarceva Taxol Taxotere Temodar Thyrogen Toposar Trelstar Depot Trelstar LA Trisenox VePesid Vinblastine Vincasar Vumon Xeloda Zanosar Zoladex Zometa Contraceptives Depo-Provera Crohn s Disease Remicade Dystonia Botox Myobloc Factor IX Concentrates Alphanine SD Benefix Mononine Profilnine SD Proplex T Bebulin VH Gaucher s Disease Ceredase Cerezyme Zavesca Growth Hormone Deficiency Genotropin Humatrope Norditropin Nutropin Nutropin AQ Saizen Hematologics Arixtra Aranesp Epogen Fragmin Innohep Lovenox Neulasta Neumega Neupogen Procrit Hepatitis C Copegus Infergen Intron A Pegasys Peg-Intron Rebetron Roferon-A Hormone Deficiency Delatestryl Delestrogen Depo-Estradiol Depo-Testosterone Hunter Syndrome Elaprase Primary Immunodeficiency Carimune NF Gamimune N Gammagard S/D Gammar-P Gamunex Iveegam EN Panglobulin Panglobulin NF Polygam S/D Venoglobulin-S Miscellaneous Alferon-N Milrinone Zinecard Multiple Sclerosis Avonex Betaseron Copaxone Novantrone Rebif Tysabri Osteo/ Rheumatoid/ Psoriatic Arthritis Enbrel Humira Hyalgan Kineret Orencia Remicade Supartz Synvisc Osteoporosis Forteo Miacalcin Psoriasis Amevive Enbrel Raptiva Remicade Respiritory Syncytial Virus Synagis Rh Hemolytic Disease BayRho-D Micro-Rhogam Rhogam WinRho-SDF