Company (location) |
Product |
Description |
Indication |
Status |
Date |
Autoimmune | |||||
Allergan plc (Dublin) and Paratek Pharmaceuticals Inc. (Boston) |
Seysara |
Sarecycline |
Moderate to severe acne vulgaris |
The FDA has accepted a new drug application |
12/22/17 |
EMD Serono Canada (Mississauga, Ontario: unit of Merck KGaA) |
Mavenclad |
Cladribine tablets |
Relapsing-remitting multiple sclerosis |
Health Canada has approved it |
12/5/17 |
Glaxosmithkline plc (London) |
Nucala |
Mepolizumab |
Eosinophilic granulomatosis with polyangiitis |
The FDA expanded the approved use and granted priority review and orphan designation |
12/13/17 |
Medday Pharmaceutical SAS (Paris) |
Qizenday |
High-dose biotin |
Progressive multiple sclerosis |
Withdrew its application following feedback from the EMA's Committee for Medicinal Products for Human Use (CHMP), which did not consider its efficacy or safety data sufficiently robust to warrant an approval |
12/18/17 |
Merck KGaA (Darmstadt, Germany) |
Mavenclad |
Cladribine tablets |
Relapsing-remitting multiple sclerosis |
Australia's Therapeutic Goods Administration updated the registration, including the indication, dosing and safety information; it is now approved to treat relapsing-remitting MS to reduce the frequency of clinical relapses and to delay the progression of physical disability |
12/8/17 |
Novartis AG (Basel, Switzerland) |
Gilenya |
Fingolimod |
Relapsing multiple sclerosis |
The FDA granted breakthrough therapy designation for the treatment of children and adolescents, 10 and older |
12/19/17 |
Pfizer Inc. (New York) |
Ixifi |
Infliximab-qbtx |
Rheumatoid arthritis, Crohn's disease, pediatric Crohn's disease, ulcerative colitis, ankylosing spondylitis, psoriatic arthritis and plaque psoriasis |
The FDA approved it |
12/15/17 |
Sanofi Genzyme (unit of Paris-based Sanofi SA) |
Dupixent |
Dupilumab |
Moderate to severe atopic dermatitis |
Health Canada has approved it |
12/6/17 |
Cancer | |||||
Agios Pharmaceuticals Inc. (Cambridge, Mass.) |
AG-120 |
Ivosidenib |
Relapsed or refractory acute myeloid leukemia |
Submitted a new drug application to the FDA |
12/28/17 |
Aptose Biosciences Inc. (San Diego and Toronto) |
CG'806 |
Pan-FLT3/pan-BTK inhibitor |
Acute myeloid leukemia |
The FDA granted orphan drug designation |
12/28/17 |
Arog Pharmaceuticals Inc. (Dallas) |
Crenolanib |
Oral benzimidazole type I kinase inhibitor |
FLT3 mutation-positive relapsed or refractory acute myeloid leukemia (AML) |
The FDA granted fast track designation |
12/4/17 |
Astrazeneca plc (Cambridge, Mass.) |
Tagrisso |
Osimertinib |
Metastatic non-small-cell lung cancer |
The FDA accepted a supplemental NDA for the use in the first-line treatment of patients whose tumors have EGFR mutations (exon 19 deletions or exon 21 [L858R] substitution mutations) and granted the application priority status |
12/19/17 |
Athenex Inc. (Buffalo, N.Y.) |
Oraxol |
Oral formulation of paclitaxel |
Paclitaxel-responsive cancers |
The U.K. Medicines and Healthcare Products Regulatory Agency granted a Promising Innovative Medicine designation |
12/28/17 |
Avillion LLP (London) and Pfizer Inc. (New York) |
Bosulif |
Bosutinib |
Newly diagnosed chronic phase Philadelphia chromosome-positive chronic myelogenous leukemia |
The FDA approved a supplemental new drug application |
12/21/17 |
Bristol-Myers Squibb Co. (New York) |
Opdivo |
Nivolumab |
Previously treated advanced or metastatic non-small-cell lung cancer |
The China FDA accepted its BLA |
12/4/17 |
Bristol-Myers Squibb Co. (New York) |
Yervoy |
Ipilimumab |
Unresectable or metastatic melanoma |
The EMA's Committee for Medicinal Products for Human Use recommended the approval of it for pediatric patients 12 years and older |
12/19/17 |
Bristol-Myers Squibb Co. (New York) |
Opdivo |
Nivolumab |
Melanoma |
The FDA approved it for intravenous use for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection |
12/21/17 |
Cellprotect Nordic Pharmaceuticals AB (Stockholm) |
Cellprotect |
Cell therapy |
Multiple myeloma |
Received EMA orphan drug status |
12/18/17 |
Celltrion Inc. (Incheon, South Korea) |
Herzuma |
Trastuzumab biosimilar |
Breast and gastric cancers |
The EMA's Committee for Medicinal Products for Human Use issued a positive opinion recommending that it be granted marketing authorization in the European Union for the treatment of patients with early breast cancer, metastatic breast cancer or metastatic gastric cancer whose tumors have either HER2 overexpression or HER2 gene amplification |
12/19/17 |
Chugai Pharmaceutical Co. Ltd. (Tokyo) |
Alecensa |
Alectinib |
Anaplastic lymphoma kinase (ALK)-positive advanced non-small-cell lung cancer (NSCLC) and ALK-positive, metastatic NSCLC |
Received approval from the European Commission; EU marketing authorization for it has moved forward to full approval to treat those with ALK-positive, metastatic NSCLC who have progressed on or are intolerant to Xalkori |
12/22/17 |
Clovis Oncology Inc. (Boulder, Colo.) |
Rubraca |
Rucaparib |
Recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer |
The FDA accepted its supplemental NDA and granted priority review status with a PDUFA date of April 6, 2018 |
12/7/17 |
Delmar Pharmaceuticals Inc. (Vancouver, British Columbia, and Menlo Park, Calif.) |
VAL-083 |
Small-molecule chemotherapeutic |
Recurrent glioblastoma |
Received fast track designation from the FDA |
12/28/17 |
Eisai Co. Ltd. (Tokyo) |
Halaven |
Eribulin |
Locally advanced or metastatic breast cancer |
The CFDA accepted for review the resubmitted NDA |
12/1/17 |
Eisai Co. Ltd. (Tokyo) |
Lenvima |
Lenvatinib mesylate |
Hepatocellular carcinoma |
Submitted an application for the additional indication |
12/7/17 |
Eisai Co. Ltd. (Tokyo) |
Halaven |
Eribulin |
Locally advanced or metastatic breast cancer |
Received a recommendation from the National Institute of Health and Care Excellence in the European Union |
12/12/17 |
Exelixis Inc. (South San Francisco) |
Cabometyx |
Cabozantinib tablets |
Advanced renal cell carcinoma |
The FDA approved it for the expanded indication |
12/20/17 |
Foundation Medicine Inc. (Cambridge, Mass.) |
Foundation Onecdx |
Pan-cancer detection assay |
Solid tumors |
The FDA approved it |
12/4/17 |
Genentech (South San Francisco, member of the Roche Group) |
Perjeta |
Pertuzumab |
HER2-positive early breast cancer |
The FDA approved it when combined with Herceptin and chemotherapy (the Perjeta-based regimen), for adjuvant treatment, post-surgery when at high risk of recurrence; the FDA also fully approved the Perjeta-based regimen for neoadjuvant treatment of HER2-positive, locally advanced, inflammatory, or early stage breast cancer (either > 2 cms in diameter or node-positive) |
12/22/17 |
Genentech Inc. (South San Francisco; member of the Roche Group) |
Avastin |
Bevacizumab |
Glioblastoma |
Gained full FDA approval |
12/7/17 |
Immunocore Ltd. (Oxford, U.K., and Conshohocken, Pa.) |
IMCgp-100 |
Targets the melanoma-associated antigen gp100 |
Metastatic uveal melanoma |
Received promising innovative medicines, or PIM, designation by the U.K.'s Medicines and Healthcare Products Regulatory Agency |
12/12/17 |
Janssen Biotech Inc. (Horsham, Pa., unit of Johnson & Johnson) |
Apalutamide |
Oral androgen receptor inhibitor |
Non-metastatic castration-resistant prostate cancer |
The FDA granted a priority review for its NDA |
12/22/17 |
Loxo Oncology Inc. (Stamford, Conn.) |
LOXO-101 |
Larotrectinib |
Unresectable or metastatic solid tumors with NTRK-fusion proteins |
Initiated the submission of an FDA rolling new drug application |
12/21/17 |
Merck & Co. Inc. (Kenilworth, N.J.) |
Keytruda |
Pembrolizumab |
Relapsed or refractory primary mediastinal large B-cell lymphoma |
The FDA accepted the supplemental biologics license application and assigned priority review |
12/12/17 |
Merck KGaA (Darmstadt, Germany) and Pfizer Inc. (New York) |
Aveluma |
PD-L1 inhibitor |
Advanced renal cell carcinoma |
The FDA granted breakthrough therapy designation for it in combination with Inlyta for treatment-naïve patients |
12/22/17 |
Mylan NV (Hertfordshire, U.K.) |
Ogivri |
Trastuzumab-dkst |
Breast or metastatic stomach cancer whose tumors overexpress the HER2 gene |
The FDA approved it |
12/4/17 |
Mylan NV (Hertfordshire, U.K.) and Biocon Ltd. (Bengaluru) |
Trastuzumab and pegfilgrastim |
Biosimilars |
HER2-positive breast and gastric cancers; neutropenia |
The EMA accepted for review its marketing authorization applications |
12/1/17 |
Novartis AG (Basel, Switzerland) |
Rydapt |
Midostaurin |
FLT3 mutated acute myeloid leukemia |
Health Canada approved it |
12/6/17 |
Novartis AG (Basel, Switzerland) |
Tafinlar and Mekinist |
Dabrafenib and trametinib |
Stage III melanoma with BRAF V600E or V600K mutations following complete resection |
The FDA has accepted the company's supplemental new drug application for filing, and granted priority review designation |
12/27/17 |
Novartis AG (Basel, Switzerland) |
Tasigna |
Nilotinib |
Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase (Ph+ CML-CP) |
The FDA approved an updated label to include treatment-free remission (TFR) data in patients after achieving sustained deep molecular response of MR4.5 |
12/28/17 |
Oncolytics Biotech Inc. (Calgary, Alberta) |
Reolysin |
Pelareorep |
Hormone receptor-positive, HER2 receptor-negative metastatic breast cancer |
Received a final advice letter from the EMA; the favorable letter suggests that a single 400-patient study may be acceptable to form the basis of a marketing authorization application in Europe |
12/6/17 |
Pharmamar SA (Madrid) |
Aplidin |
Plitidepsin |
Multiple myeloma |
The EMA's Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion in response to its application for approval |
12/18/17 |
Puma Biotechnology Inc. (Los Angeles) |
Neratinib |
Irreversible tyrosine kinase inhibitor |
Adjuvant early stage HER2-positive breast cancer |
The EMA has requested that the Scientific Advisory Group on Oncology provide an opinion on the clinical aspects of the marketing authorization application (MAA) for it at a meeting to be held on Jan. 11, 2018 |
12/15/17 |
Rhizen Pharmaceuticals SA (La Chaux-de-Fonds, Switzerland) |
RP-6530 |
Tenalisib |
Relapsed/refractory peripheral T-cell lymphoma |
The FDA granted fast track designation |
12/12/17 |
Rhizen Pharmaceuticals SA (La Chaux-de-Fonds, Switzerland) |
RP-6530 |
Tenalisib |
Peripheral T-cell lymphoma |
Received FDA orphan drug designation |
12/27/17 |
Samsung Bioepis Co. Ltd. (Incheon, Korea) |
SB-3 |
Biosimilar candidate referencing Herceptin |
Early breast cancer, metastatic breast cancer and metastatic gastric cancer |
The FDA accepted its BLA application under the 351(k) pathway |
12/21/17 |
Shire plc (Dublin) |
Oncaspar |
Pegaspargase |
Acute lymphoblastic leukemia |
The European Commission granted marketing authorization |
12/14/17 |
Sirnaomics Inc. (Gaithersburg, Md.) |
STP-705 |
Small interfering RNA therapeutic |
Cholangiocarcinoma |
The FDA granted orphan drug designation |
12/11/17 |
Cardiovascular | |||||
Amgen Inc. (Thousand Oaks, Calif.) |
Repatha |
Evolocumab |
Prevention of heart attacks, strokes and coronary revascularizations |
Following priority review of its supplemental biologics license application, the FDA approved it |
12/5/17 |
Carmel Biosciences Inc. (Atlanta) |
Prexxartan |
Valsartan oral solution |
Hypertension and heart failure |
The FDA approved it |
12/21/17 |
Janssen Research and Development LLC (Raritan, N.J.) |
Xarelto |
Rivaroxaban |
Vascular indications |
Submitted a supplemental new drug application to the FDA for two new indications: reducing the risk of major cardiovascular events such as death, heart attack or stroke in patients with chronic coronary and/or peripheral artery disease (PAD), and for reducing the risk of acute limb ischemia in patients with PAD |
12/12/17 |
Mesoblast Ltd. (Melbourne, Australia) |
MPC |
Mesenchymal precursor cell therapy |
Heart failure |
The FDA granted regenerative medicine advanced therapy designation for the treatment of patients with left ventricular systolic dysfunction and left ventricular assist devices (LVADs) |
12/22/17 |
Portola Pharmaceuticals Inc, (South San Francisco) |
Andexxa |
Andexanet alfa |
Reversal of the anticoagulant effects of apixaban and rivaroxaban in major bleeding |
The FDA extended the review of the BLA by 90 days after the company submitted additional data from the ongoing ANNEXA-4 study that was requested by the agency; the new Prescription Drug User Fee Act action date is May 4, 2018 |
12/28/17 |
Portola Pharmaceuticals Inc. (South San Francisco) |
Bevyxxa |
Betrixaban |
Prevention of venous thromboembolism |
The EMA's Committee for Medicinal Products for Human Use requested additional information related to the marketing authorization application (MAA) |
12/13/17 |
Renova Therapeutics Inc. (San Diego) |
RT-100 |
AC6 gene transfer (Ad5.hAC6) |
Heart failure with reduced ejection fraction |
The FDA gave fast track designation |
12/15/17 |
Steadymed Ltd. (San Ramon, Calif.) |
Trevyent |
Treprostinil, delivered by Patchpump technology |
Pulmonary arterial hypertension |
After a refusal-to-file letter in August, the FDA provided guidance at a type A meeting about what is needed to resubmit the NDA; it asked the company to repeat in vitro design verification (DV) testing on the final to-be-marketed product |
12/11/17 |
Central Nervous System | |||||
Acorda Therapeutics Inc. (Ardsley, N.Y.) |
Inbrija |
Inhaled levodopa |
Parkinson's disease |
Resubmitted its NDA, addressing the issues raised in the recent refuse-to-file letter – the date on which the manufacturing site will be ready for inspection and a question regarding the submission of the drug master production record – and includes all additional information requested by the FDA |
12/8/17 |
Aptinyx Inc. (Evanston, Ill.) |
NYX-783 |
Modulates N-methyl-D-aspartate receptors |
Post-traumatic stress disorder |
The FDA granted fast track designation |
12/8/17 |
Concentric Analgesics Inc. (Los Altos, Calif.) |
CA-008 |
Water-soluble prodrug that converts into capsaicin |
Postsurgical pain |
The FDA granted fast track designation |
12/8/17 |
Egalet Corp. (Wayne, Pa.) |
Arymo ER |
Morphine sulfate extended-release tablets C-II |
Pain |
The FDA granted tentative approval for an expanded label for the management of pain severe enough to require daily, around-the-clock, long-term opioid treatment and for which alternative treatment options are inadequate; final approval is expected to be granted when the exclusivity period expires on Oct. 2, 2018 |
12/18/17 |
Eisai Co. Ltd. (Tokyo) |
Aricept |
Donepezil |
Severe Alzheimer's disease |
Was approved for the additional indication in China |
12/14/17 |
GW Pharmaceuticals plc ( London) |
Epidiolex |
Cannabidiol |
Seizures associated with Lennox-Gastaut syndrome and Dravet syndrome |
The FDA accepted for filing with priority review its new drug application, setting a PDUFA date of June 27, 2018 |
12/29/17 |
H. Lundbeck A/S (Valby, Denmark) |
Brintellix |
Vortioxetine |
Depression |
The China FDA approved it |
12/5/17 |
Indivior (Slough, U.K.) |
Sublocade |
Buprenorphine extended-release |
Moderate to severe opioid use disorder |
The FDA approved it |
12/4/17 |
Indivior plc (Slough, U.K.) |
RBP-7000 |
Once-monthly injectable risperidone using the Atrigel delivery system |
Schizophrenia |
The FDA accepted the new drug application, setting a PDUFA date of July 28, 2018 |
12/13/17 |
Insys Therapeutics Inc. (Phoenix) |
Buprenorphine |
Sublingual spray |
Moderate to severe acute pain |
Its NDA was accepted for filing by the FDA with a PDUFA date of July 28, 2018 |
12/7/17 |
Kitov Pharmaceuticals Ltd. (Tel Aviv, Israel) |
KIT-302 |
Celecoxib and amlodipine besylate |
Pain caused by osteoarthritis and hypertension |
The FDA granted permission for the firm to use the brand name Consensi for marketing, subject to receipt of marketing approval |
12/15/17 |
Lannett Company Inc. (Philadelphia) |
Numbrino |
Cocaine hydrochloride (HCl) topical solution, 4% and 10% |
Local anesthesia for diagnostic procedures and surgeries |
Its 505(b)(2) new drug application has been accepted for filing by the FDA with a PDUFA date of July 21, 2018 |
12/4/17 |
Ovid Therapeutics Inc. (New York) |
TAK-935/OV-935 |
Cholesterol 24-hydroxylase inhibitor |
Dravet syndrome |
The FDA granted orphan drug designation |
12/6/17 |
Ovid Therapeutics Inc. (New York) |
TAK-935/OV-935 |
Inhibitor of cholesterol 24-hydroxylase |
Lennox-Gastaut syndrome |
The FDA granted orphan drug designation |
12/21/17 |
Teva Pharmaceutical Industries Ltd. (Jerusalem) |
Fremanezumab |
Anti-calcitonin gene-related peptide monoclonal antibody |
Migraine prevention |
The FDA accepted its BLA; the company used a priority review voucher |
12/19/17 |
Teva Pharmaceutical Industries Ltd. (Jerusalem) |
Fremanezumab |
Anti-calcitonin gene-related peptide monoclonal antibody |
Cluster headache prevention |
The FDA granted fast track designation |
12/19/17 |
Diabetes | |||||
Merck & Co. Inc. (Kenilworth, N.J.) and Pfizer Inc. (New York) |
Ertugliflozin |
SGLT2 inhibitor |
Type 2 diabetes |
The FDA approved it |
12/22/17 |
Novo Nordisk A/S (Bagsvaerd, Denmark) |
Ozempic |
Semaglutide |
Type 2 diabetes |
The FDA approved it |
12/6/17 |
Novo Nordisk A/S (Bagsvaerd, Denmark) |
Ozempic |
Semaglutide |
Type 2 diabetes |
The EMA's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a marketing authorization |
12/18/17 |
Sanofi SA (Paris) |
Admelog |
Insulin lispro injection |
Type 1 and type 2 diabetes |
The FDA approved it for adults and pediatric patients ages 3 and older with type 1 diabetes mellitus and in adults with type 2 diabetes mellitus; it was approved as a follow-on product submitted through the agency's 505(b)(2) pathway |
12/12/17 |
Gastrointestinal | |||||
Pfizer Inc. (New York) |
Xeljanz |
Tofacitinib |
Moderately to severely active ulcerative colitis |
The FDA extended the action date by three months for the supplemental NDA; the agency determined additional review time was needed due to information recently submitted that constitutes a major amendment; the PDUFA date is in June 2018 |
12/14/17 |
Seres Therapeutics Inc. (Cambridge, Mass.) |
SER-287 |
Microbiome therapeutic candidate |
Ulcerative colitis |
Gained FDA orphan drug status |
12/5/17 |
Shire plc (Dublin) |
SHP-647 |
Human IgG2 monoclonal antibody |
Moderately to severely active ulcerative colitis |
Gained FDA orphan drug status |
12/1/17 |
Tigenix NV (Leuven, Belgium) |
Alofisel |
Darvadstrocel |
Complex anal fistulas caused by Crohn's disease |
The EMA's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a marketing authorization |
12/18/17 |
Infection | |||||
60 Degrees Pharmaceuticals LLC (Washington) |
60P003 |
Tafenoquine |
Malaria |
Submitted an NDA to the FDA |
12/19/17 |
Basilea Pharmaceutica Ltd. (Basel, Switzerland) |
Ceftobiprole |
Cephalosporin antibiotic |
Bacterial bloodstream infections (bacteremia) caused by Staphylococcus aureus |
The FDA has given it a qualified infectious disease product designation |
12/22/17 |
La Jolla Pharmaceutical Co. (San Diego) |
Giapreza |
Angiotensin II |
Septic or distributive shock |
The FDA approved it |
12/27/17 |
Medimetriks Pharmaceuticals Inc. (Fairfield, N.J.) |
Xepi |
Ozenoxacin cream, 1% |
Impetigo |
The FDA approved it for patients 2 months of age and older when applied topically twice daily for five days |
12/15/17 |
Siga Technologies Inc. (New York) |
TPOXX |
Tecovirimat, oral formulation |
Smallpox |
Submitted its new drug application to the FDA |
12/12/17 |
Starpharma Ltd. (Melbourne) |
Vivagel |
Mucoadhesive gel |
Recurrent bacterial vaginosis |
Submitted a rolling submission of its new drug application to the FDA |
12/1/17 |
Inflammatory | |||||
Eli Lilly and Co. (Indianapolis) |
Taltz |
Ixekizumab 80 mg/mL |
Active psoriatic arthritis |
The FDA approved it |
12/5/17 |
Pfizer Inc. (New York) |
Xeljanz and Xeljanz XR |
Tofacitinib, 5 mg twice daily and 11 mg once daily |
Active psoriatic arthritis |
The FDA approved both for adults who have had an inadequate response or intolerance to methotrexate or other disease-modifying antirheumatic drugs |
12/18/17 |
Miscellaneous | |||||
Achillion Pharmaceuticals Inc. (New Haven, Conn.) |
ACH-4471 |
Small-molecule drug designed to inhibit complement factor D |
C3 glomerulopathy |
The FDA granted orphan designation |
12/19/17 |
Aclaris Therapeutics Inc. (Malvern, Pa.) |
Eskata |
Hydrogen peroxide) topical solution, 40% |
Raised seborrheic keratoses |
The FDA approved it |
12/18/17 |
Aerie Pharmaceuticals Inc. (Irvine, Calif.) |
Rhopressa |
Netarsudil ophthalmic solution, 0.02% |
Open-angle glaucoma or ocular hypertension |
The FDA approved it |
12/19/17 |
Aeterna Zentaris Inc. (Charleston, S.C.) |
Macrilen |
Macimorelin |
Adult growth hormone deficiency |
The FDA granted marketing approval |
12/22/17 |
Agile Therapeutics Inc. (Princeton, N.J.) |
Twirla |
Non-daily, low-dose combination hormonal contraceptive patch |
Prevention of pregnancy |
The FDA issued a complete response letter (CRL) in response to the NDA identifying deficiencies relating to quality adhesion test methods and noted that observations identified during an inspection of a facility of the company's third-party manufacturer, Corium International Inc., must be resolved; the CRL recommends that the company address the implications of clinical trial subject patch compliance and the withdrawal and dropout rates |
12/27/17 |
Alexion Pharmaceuticals Inc. (New Haven, Conn.) |
Soliris |
Eculizumab |
Generalized myasthenia gravis |
Received approval in Japan as a treatment for patients who are anti-acetylcholine receptor (AChR) antibody-positive and whose symptoms are difficult to control with high-dose intravenous immunoglobulin (IVIG) therapy or plasmapheresis (PLEX) |
12/28/17 |
Alnylam Pharmaceuticals Inc. (Cambridge, Mass.) |
Patisiran |
RNA interference therapeutic targeting transthyretin |
Transthyretin-mediated amyloidosis |
The FDA granted a request to amend the orphan drug designation |
12/12/17 |
Alnylam Pharmaceuticals Inc. (Cambridge, Mass.) |
Patisiran |
RNA interference therapeutic targeting transthyretin |
ATTR amyloidosis |
Completed the rolling submission of a new drug application and requested a priority review |
12/13/17 |
Amicus Therapeutics Inc. (Cranbury, N.J.) |
Galafold |
Migalastat |
Fabry disease |
Submitted an NDA to the FDA requesting approval for treating patients, 16 and older, with Fabry disease who have amenable mutations |
12/15/17 |
Antares Pharma Inc. (Ewing, N.J.) |
Xyosted |
Testosterone enanthate |
Hypogonadism |
A written request for a type A meeting, along with a comprehensive briefing document, have been submitted to the FDA, in response to the complete response letter regarding the NDA |
12/27/17 |
Armagen Inc. (Calabasas, Calif.) |
AGT-181 |
Enzyme replacement therapy |
Hurler syndrome |
The FDA granted fast track designation |
12/1/17 |
Biogen Inc. (Cambridge, Mass.) |
Spinraza |
Nusinersen |
5q spinal muscular atrophy |
Received a recommendation by the Canadian Drug Expert Committee for approval to treat in a subset population of patients most likely to have or to develop Type 1 disease and diagnosed within a narrow clinical window |
12/28/17 |
Biomarin Pharmaceutical Inc. (San Rafael, Calif.) |
Pegvaliase |
Pegylated recombinant phenylalanine ammonia lyase enzyme product |
Phenylketonuria |
The FDA will require additional time to complete its review of the BLA; the PDUFA date was extended to May 25, 2018 |
12/27/17 |
Biovie Inc. (Beverly, Mass.) |
BIV-201 |
Continuous infusion terlipressin |
Refractory ascites due to liver cirrhosis |
Granted fast track designation by the FDA |
12/12/17 |
Diurnal Ltd. (Cardiff, U.K.) |
Alkindi |
Hydrocortisone |
Adrenal insufficiency |
Approved under the EMA's pediatric-use marketing authorization (PUMA) pathway |
12/18/17 |
Innovus Pharmaceuticals Inc. (San Diego) |
Prostagorx |
Multiresponse prostate supplement |
Benign prostatic hyperplasia |
Filed a natural health product license application in Canada |
12/5/17 |
Insys Therapeutics Inc. (Phoenix) |
CBD |
Cannabidiol oral solution |
Prader-Willi syndrome |
The FDA granted fast track designation |
12/28/17 |
Jazz Pharmaceuticals plc (Dublin) |
JZP-110 |
Solriamfetol |
Excessive sleepiness |
Submitted its NDA to the FDA |
12/22/17 |
Kyowa Hakko Kirin Co. Ltd. (Tokyo) |
Crysvita |
Burosumab |
X-linked hypophosphatemia |
The EMA's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a marketing authorization |
12/18/17 |
Medicines Development for Global Health (Australia, not-for-profit company) |
Moxidectin |
Semi-synthetic methoxime derivative of LL F-2924α |
Onchocerciasis |
Submitted an NDA; the FDA confirmed the application qualified for priority review |
12/21/17 |
Meiragtx Ltd. (London) |
A-001 |
Gene therapy |
Leber's congenital amaurosis due to mutations in the RPE65 gene (LCA2) |
The Offices of Orphan Products Development and Pediatric Therapeutics of the FDA have granted rare pediatric disease designation |
12/8/17 |
Neurovive Pharmaceutical AB (Lund, Sweden) |
KL-1333 |
Modulator of the cellular levels of NAD+ |
Genetic mitochondrial diseases |
The European Commission granted orphan designation for it for oral treatment of mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like episodes, or MELAS |
12/14/17 |
Omeros Corp. (Seattle) |
Omidria |
Phenylephrine and ketorolac intraocular solution, 1%/0.3% |
Prevention of intraoperative miosis |
The FDA approved the supplemental new drug application, which expands the indication to include use in pediatric patients (ages birth through 17 years) |
12/13/17 |
Organovo Holdings Inc. (San Diego) |
3D bioprinted liver therapeutic tissue |
Tissue therapy |
Alpha-1 antitrypsin deficiency |
The FDA granted orphan drug designation |
12/28/17 |
Ovid Therapeutics Inc. (New York) |
OV-101 |
Delta-selective GABAA receptor agonist |
Angelman syndrome |
The FDA granted fast track designation |
12/20/17 |
Protalix Biotherapeutics Inc. (Carmiel, Israel) |
PRX-102 |
Pegunigalsidase alfa |
Fabry disease |
The European Commission granted orphan drug designation |
12/28/17 |
Regeneron Pharmaceuticals Inc. (Tarrytown, N.Y.) |
Eylea |
Aflibercept injection |
Wet age-related macular degeneration |
The FDA accepted for review its supplemental BLA for a 12-week dosing interval based on physician's assessment; the PDUFA date is Aug. 11, 2018 |
12/12/17 |
Sangamo Therapeutics Inc. (Richmond, Calif.) |
SB-318 and SB-913 |
Genome editing candidates |
Rare lysosomal storage disorders mucopolysaccharidosis type I (MPS I) and MPS II |
The EMA issued a positive opinion on the application for orphan medicinal product designation |
12/11/17 |
Sanofi SA (Paris, Sanofi Genzyme unit) and Alnylam Pharmaceuticals Inc. (Cambridge, Mass.) |
Patisiran |
RNAi therapeutic targeting transthyretin |
Hereditary transthyretin-mediated amyloidosis |
Submitted an MAA to the EMA |
12/19/17 |
Santen Pharmaceutical Co. Ltd. (Osaka, Japan) and its subsidiary Santen Inc. (Emeryville, Calif.) |
DE-109 |
Intravitreal sirolimus |
Noninfectious uveitis of the posterior segment |
The FDA issued a complete response letter for the NDA and requested additional substantiating evidence to demonstrate efficacy |
12/22/17 |
Spark Therapeutics Inc. (Philadelphia) |
Luxturna |
Voretigene neparvovec-rzyl |
Biallelic RPE65 mutation-associated retinal dystrophy |
The FDA approved it |
12/20/17 |
Spruce Biosciences Inc. (San Francisco) |
SPR-001 |
Small molecule |
Congenital adrenal hyperplasia |
The FDA has granted orphan drug status |
12/5/17 |
Stealth Biotherapeutics Inc. (Boston) |
Elamipretide |
Mitochondria-targeting peptide |
Leber's hereditary optic neuropathy |
The FDA granted fast track designation |
12/19/17 |
Therapeuticsmd Inc. (Boca Raton, Fla.) |
TX-004HR |
Applicator-free estradiol vaginal softgel capsule |
Moderate to severe vaginal pain during sexual intercourse |
Resubmitted its new drug application to the FDA |
12/1/17 |
Wilson Therapeutics AB, (Stockholm) |
WTX-101 |
Bis-choline tetrathiomolybdate |
Wilson disease |
The FDA granted fast track designation |
12/15/17 |
Respiratory | |||||
Adamis Pharmaceuticals Corp. (San Diego) |
Symjepi |
Epinephrine injection |
Allergic reactions (type I) |
Submitted a prior approval supplement to the FDA for a pediatric version (0.15 mg), approved at the 0.3-mg dose for the emergency treatment of allergic reactions (type I), including anaphylaxis, designed for patients weighing 66 pounds or greater |
12/1/17 |
ALK-Abelló A/S (Copenhagen) |
Acarizax |
Aublingual allergy immunotherapy |
Allergic rhinitis and allergic asthma |
Reached a satisfactory agreement with health authorities in France for the pricing and reimbursement |
12/1/17 |
Prometic Life Sciences Inc. (Laval, Quebec) |
PBI-4050 |
Orally active drug candidate |
Idiopathic pulmonary fibrosis |
The U.K.'s Medicines and Healthcare Products Regulatory Agency issued a promising innovative medicine, or PIM, designation as add-on treatment to nintedanib |
12/12/17 |
Prometic Life Sciences Inc. (Laval, Quebec) |
Ryplazim |
Plasminogen |
Idiopathic pulmonary fibrosis |
The FDA granted orphan designation |
12/19/17 |
Sunovion Pharmaceuticals Inc. (Marlborough, Mass.) |
Lonhala Magnair |
Glycopyrrolate inhalation solution |
Chronic obstructive pulmonary disease |
The FDA approved it |
12/7/17 |
Notes The date indicated refers to the BioWorld issue in which the news item can be found. For more information about individual companies and/or products, see Cortellis. |