Reviewing the drug class of 2011

Inside BIO Industry Analysis

The drug class of 2011 was larger than the past few graduating classes. We count over 30 new “innovative” drugs found among the list of FDA approvals last year. This is in contrast to the 20-something range of the past few years. On the list are some clear breakthrough treatments and a few glimpses of “the future of biotech”. The specific drugs are listed further below, but first a big picture view:

Ten of the approvals were for biologics, six of which came through CDER. Of these six, three were antibodies, two were recombinant fusions, and one was a PEG linked enzyme. The other four biologics came through CBER. The CBER approvals contain blood or serum derived therapies such as the first blood cord stem cell therapy (Hemacord), a mixture of horse derived antibody fab domains for scorpion venom (Anascorp), and an autologous cell therapy for wrinkles (LaViv). One recombinant blood protein was approved by CBER, rFactor XIII (Corifact).

A range of disease areas were addressed by the the approved therapies. Cancer, being the most prevalent with eight approvals. Two of the small molecule new molecular entities (NMEs) are kinase inhibitors for cancers that were approved along side companion diagnostics. We expect to see more of this in the future, as patient identification and responder selection gains more traction in the industry, and regulators welcome the concept. News this week from Biogen, regarding Tysabri, underscores the importance using diagnostics for patient stratification even for approved drugs. News that Roche has bid ~$6B for Illumina also illustrates the imperative for drug developers. Examples of companion diagnostic for negative/positive responder identification, as well as safety profiling, will likely become a major theme in biotech in coming years.

The drugs slated for fast review and decision accounted for over 50% of the approvals. Orphan designated drugs made up a fifth of these approvals, and over a third were priority approvals.

Counting approvals that are new and “innovative”, is not as straight forward as copying and pasting from fda.gov. First, we only include drugs that are being approved for the first time, for a first indication. Second, we do not include imaging agents in our list, like the FDA did in their fiscal year list back in November (note we use a calendar year here). Third, unlike lists published elsewhere online that only count CDER BLA approvals, we include certain BLAs approved via CBER. Fourth, we exclude new formulations, and combinations. However, we do include chemically modified forms of exisiting drugs, such as this year’s addition of PEG to arginase in Erwinase (chemically like what Amgen did for Neulasta or Celltech did for Cimzia). As another example, two drugs with Gabapentin were approved in 2011, but only one was chemically unique (the prodrug Horizant) and made it on our list. The other, Gralise is a once daily reformulation.

Below is the 2011 list of new drugs. The colors denote biologics: Antibody (green), fusion or native protein (blue), blood fraction or cell therapy (orange).

Approval Date FDA’s Class Product Name Active Ingredient Company (partner or acquirer) Indication & Significance
1 1/18/2011 NME Natroba SPINOSYN/ SPINOSAD PARAPRO PHARM Topical treatment for head lice. The drug is an insecticide originally isolated from a soil dwelling bacteria
2 1/21/2011 NME Viibryd VILAZODONE HCL Clinical Data (Forest Labs) Treats clinical depression, with less side effects than other anti-depressants. Novel dual mechanism of action as both a potent and selective serotonin reuptake inhibitor (SSRI) and a partial agonist of the 5-HT1A receptor.
3 2/25/2011 NME Edarbi AZILSARTAN MEDOXOMIL Takeda Treats high blood pressure. More effective in lowering 24-hour blood pressure compared with Diovan and Benicar. It is an angiotensin II receptor antagonist.
4 2/11/2011 BLA-CBER Corifact rFactor XIII CSL Behring 1st product intended to prevent bleeding in people with congenital factor XIII deficiency. (Already approved in 12 other countries as Fibrogammin® P)
5 3/7/2011 NME Daxas Roflumilast Forest Labs, Nycomed 1st oral anti-inflammatory treatment developed specifically for COPD patients. Selective, long-acting inhibitor of PDE-4. Approved in the EU June 2010.
6 3/10/2011 BLA-CDER Benlysta Belimumab Human Genome Sciences 1st treatment for Lupus in 50 years. Fully human Mab vs. B-lymphocyte stimulator (BLyS).
7 3/16/2011 NME Ardovax Adenovirus Type 4, 7 Vaccine Live, oral Barr (Teva) Live virus, tablet for vaccination of U.S. military personnel only. Although a vaccine against adenovirus types 4 and 7 was given to U.S. military recruits from 1971 to 1999. The manufacturer stopped producing the vaccine in 1999.
8 4/1/2011 BLA-CDER Yervoy Ipilimumab BMS (originally Medarex) 1st melanoma treatment to improve overall survival. Medarex, a small biotech devloped this fully human MAb vs CTLA4.
9 4/6/2011 NME Horizant Gabapentin enacarbil Xenoport (GSK) Prodrug for the anticonvulsant and analgesic drug gabapentin (Increases bioavailablity of an existing drug.)
10 4/7/2011 NME Vandetanib ZD6474 AstraZeneca 1st drug approved for metastatic thyroid cancer for adults ineligible for surgery. Inhibits VEGFR and EGFR.
11 4/25/2011 NME Duexis Famotidine Horizon Pharma Treatment for patients with RA and OA. Combination of ibuprofen and famotidine
12 4/28/2011 NME Zytiga Abiraterone Cougar Biotech (JNJ) Therapy for castration-resistant prostate cancer. Inhibits CYP17A1, an enzyme expressed in testicular, adrenal, and prostatic tumor tissues.
13 5/2/2011 NME Tradjenta  Linagliptin Boehringer Ingelheim Type II diabetes. DPP-4 inhibitor, an enzyme that degrades the incretin hormones (GLP-1) and (GIP).
14 5/2/2011 NME Victrelis boceprevir Schering-Plough (Merck) Novel Hepatitis C drug targeting the NS3 serine protease
15 5/20/2011 NME Edurant RILPIVIRINE HCL Tibotec ( JNJ) Treatment for people starting HIV retrovirals for the first time. Second-generation non-nucleoside reverse transcriptase inhibitor (NNRTI) with higher potency, longer half-life and reduced side-effect profile
16 5/23/2011 NME Incivek telaprevir Vertex (JNJ) Novel Hepatitis C drug targeting the NS3 serine protease
17 5/30/2011 NME Dificid Fidaxomicin Optimer Pharmaceuticals New class of antibiotics. First drug in 25 years to target a specific bacteria that infects the intestines (Clostridium difficile) but leaves healthy bacteria alone. Clostridium difficile infections are commonly found in hospitals. Innhibits RNA polymerase.
18 6/13/2011 NME Potiga ezogabine Valeant (GSK) 1st in a new class of drugs for partial epileptic seizures (the most common type). Acts as a neuronal KCNQ/Kv7 potassium channel opener, a mechanism of action markedly different from than of any current anticonvulsants.
19 6/15/2011 BLA-CDER Nulojix Belatacept BMS Kidney transplant anti-rejection drug. Belatacept is a fusion of the Fc to the extracellular domain of CTLA-4.
20 6/22/2011 BLA-CBER laViv Fibroblasts, autologous Fibrocell Science 1st personalized cell therapy approved by the FDA for aesthetic use. Isolated blood cord stem cells.
21 7/1/2011 NME Xarelto Rivaroxaban Bayer(JNJ) 1st available orally active direct factor Xa inhibitor.(On July 1, 2011, the FDA approved rivaroxaban for prophylaxis of deep vein thrombosis (DVT), which may lead to pulmonary embolism (PE), in adults undergoing hip and knee replacement surgery.[2]. In Nov. 2011, it was approved for stroke prophylaxis in patients with non-valvular atrial fibrillation.
22 7/1/2011 NME Arcapta INDACATEROL MALEATE Novartis A novel once-daily bronchodilato for long-term maintenance of airflow obstruction in COPD. Ultra-long-acting beta-adrenoreceptor agonist.
23 7/20/2011 NME Brilinta TICAGRELOR AstraZeneca Blood thinner for patients with acute coronary syndrome (ACS). It is a platelet aggregation inhibitor.
24 8/3/2011 BLA-CBER Anascorp Scorpion antitoxin Rare Disease Therapeutics Inc. 1st therapeutic approved for scorpion stings. A polyvalent preparation of equine immune globulin F(ab’)2 fragments, manufactured from plasma of horses immunized with scorpion venom.
25 8/17/2011 NME Zelboraf Vemurafenib Plexxikon (Daiichi, Roche) Targets patients with the BRAF V600E mutation (identified with companion dx). Second drug approved for Melanoma in 2011. Inhibihits ser/thr kinase B-Raf .
26 8/19/2011 BLA-CDER Adcetris Brentuximab vedotin Seattle Genetics First new treatment for Hodgkin’s lymphoma in 34 years. Antibody targeting CD-30, with cytotoxin attached.
27 8/25/2011 NME Firazyr Icatibant Shire First self-injectable anti-swelling drug to treat hereditary angioedema, a rare swelling disease. A peptidomimetic (ten amino acids) bradykinin B2 receptor antagonist.
28 8/26/2011 BLA-CDER EYLEA AFLIBERCEPT REGENERON PHARMACEUTICALS Treatment of Wet Age-related Macular Degeneration (AMD). Recombinant fusion protein consisting of portions of the VEGF receptors 1 and 2 extracellular domains fused to Fc. Total size:115kD.
29 8/26/2011 NME Xalkori Crizotinib Pfizer Treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) that is ALK- positive (identified by FDA approved Companion Dx). Inhibits anaplastic lymphoma kinase.
30 8/26/2011 NME DEFERIPRONE FERRIPROX ApoPharma, Inc Approved for second-line treatment of blood transfusion- related iron overload. Oral drug that chelates iron.
31 11/10/2011 BLA-CBER Hemacord Cord blood stem cells (Hemacord) New York Blood Center, Inc. 1st cord blood therapy approved by FDA. Indicated for use in unrelated donor hematopoietic progenitor cell transplantation procedures (ex: in acute Leukemia). Contains hematopoietic progenitor cells (stem cells) in cord blood.
32 11/16/2011 NME Jakafi RUXOLITINIB PHOSPHATE Incyte Pharmaceuticals 1st and only treatment for the bone marrow disease myelofibrosis. 1st approved Janus Kinase (JAK) inhibitor.
33 11/22/2011 NME Erwinaze recombinant, pegylated Asparaginase EUSA Pharma Treatment for Acute Lymphoblastic Leukemia (ALL), the most common type of childhood cancer. This E. coli-derived aspariginase acts by catalytically depleting blood asparigine but has immunogenic advantages due to pegylating the recombinant enzyme.

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