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.