Pipeline

New Pipeline Updates from Akcea Therapeutics, Genentech, Eximo Medical and Valneva USA

October 15, 2018

Company Drug/Device Medical Condition Status
Akcea Therapeutics TEGSEDITM (inotersen) Polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults Granted approval by the FDA
Akcea Therapeutics AKCEA-APO(a)-LRx Cardiovascular disease (CVD) and elevated levels of lipoprotein(a), or Lp(a) Positive topline results from a Phase II clinical trial
Bausch Health Companies, Inc. BRYHALI™ (halobetasol propionate) Lotion, 0.01% Plaque psoriasis in adults FDA tentatively approved New Drug Application; final OK pending expiration of exclusivity for related product
Eximo Medical Ltd B-Laser™ Atherectomy System Peripheral artery disease (PAD) Granted 510(k) clearance by the FDA
YiSheng BioPharma Co. PIKA® rabies vaccine Rabies Granted clearance by the China FDA to proceed with a clinical trial
Genentech Hemlibra Hemophilia A patients without factor VIII inhibitors Granted approval by the FDA
Leadiant Biosciences, Inc. Revcovi™ (elapegademase-lvlr) injection Adenosine deaminase severe combined immune deficiency (ADA-SCID) in children and adults Granted approval by the FDA
Breckenridge Pharmaceutical, Inc. Roflumilast Tablets, 500mcg (generic for Daliresp® Tablets) COPD Abbreviated New Drug Application approved by the FDA
Valneva USA XIARO® (Japanese Encephalitis Vaccine, Inactivated, Adsorbed) Japanese encephalitis Accelerated dosing regimen approved by the FDA
Eidos Therapeutics, Inc. AG10 amyloidosis (ATTR) Granted Orphan Drug designation by the FDA
Paratek Pharmaceuticals, Inc. NUZYRA™ (omadacycline) Community-acquired bacterial pneumonia; acute skin infections (ABSSSI) in adults Granted approval by the FDA
Poxel SA Imeglimin, an investigational therapeutic agent Type 2 diabetes Patient enrollment completed in the TIMES 2 trial of the Phase III registration program
Chugai Pharmaceutical Co., Ltd HEMLIBRA® (US generic name: emicizumab-kxwh) Hemophilia A without factor VIII inhibitors in newborns, children and adults Granted approval by the FDA
Genentech baloxavir marboxil Influenza type A/H3N2 and type B Phase III CAPSTONE-2 trial showed symptoms improved significantly faster in patients at high risk of serious flu who took the drug v. a placebo
Zealand Pharma A/S Glepaglutide Short bowel syndrome (SBS) First patient enrolled in a global Phase III trial

New Pipeline Updates from Amgen, Eli Lilly, Applied BioCode and Ocugen

October 8, 2018

Company Drug/Device Medical Condition Status
Paratek Pharmaceuticals, Inc. SEYSARA™ (sarecycline) Inflammatory lesions of non-nodular moderate to severe acne vulgaris in patients 9 years of age and older Granted approval by the FDA
Clovis Oncology, Inc. Rubraca® (rucaparib) BRCA1/2-mutated mCRPC after androgen receptor (AR)-directed therapy and taxane-based chemotherapy Granted Breakthrough Therapy designation by the FDA
PharmAbcine, Inc. Bevacizumab (Avastin®) Recurrent glioblastoma Received “Study May Proceed Letter “ for IND application from the FDA; can initiate Phase II trials
Amgen KYPROLIS® (carfilzomib) Relapsed or refractory multiple myeloma FDA approved supplemental New Drug Application to expand prescribing information
Applied BioCode BioCode® Gastrointestinal Pathogen Panel on BioCode® MDx-3000 molecular system Tests for 17 most common bacteria, viruses and parasites that cause infectious diarrhea Granted 510 (k) clearance by the FDA
Eisai Co., Ltd. Fycompa (perampanel) Partial-onset seizures in pediatric patients with epilepsy 4 years of age and older Granted approval by the FDA for an indication expansion
Eli Lilly and Company Ultra Rapid Lispro (URLi) Type 1 and Type 2 diabetes Phase III clinical trials show met primary efficacy endpoint
Seattle Genetics, Inc. and Takeda Pharmaceutical Company Limited ADCETRIS (brentuximab vedotin) in combination with CHP (cyclophosphamide, doxorubicin, prednisone) CD30-expressing peripheral T-cell lymphoma (PTCL), also known as mature T-cell lymphoma (MTCL) Phase III ECHELON-2 clinical trial met its primary endpoint
Acorda Therapeutics, Inc. INBRIJA, investigational inhaled levodopa OFF periods in people with Parkinson’s disease taking a carbidopa/levodopa regimen Releasing efficacy analysis from SPAN-PD, Phase III clinical trial
Five Prime Therapeutics, Inc. Bemarituzumab (FPA144), an isoform-selective FGF receptor 2b (FGFR2b) antibody, with chemotherapy Advanced gastric cancer (GC) or gastroesophageal junction (GEJ) cancer First patient dosed in Phase III FIGHT pivotal trial
Ocugen, Inc. OCU310 Dry eye Initiated Phase III clinical trial
PTC Therapeutics Risdiplam (RG7916) Type 1, 2 and 3 spinal muscular atrophy (SMA) Interim data from open-label clinical trials show increases in developmental motor milestones
Veloce BioPharma, LLC VBP-926 (Povidone-Iodine) topical treatment Chemo-associated paronychia, or painful, inflamed nails, a chemo side effect Positive results of Phase IIb trial

New Pipeline Updates from AstraZeneca, Genentech, Cook Medical and Avita Medical

October 1, 2018

Company Drug/Device Medical Condition Status
Verastem Copiktra Leukemia Granted approval by the FDA
MT.DERM GmbH Exceed, microneedling device Facial wrinkles Granted clearance by the FDA
Cook Medical 5 mm diameter version of Zilver® PTX® Peripheral artery disease (PAD) Granted clearance by the FDA
Avita Medical RECELL® Autologous Cell Harvesting Device (RECELL® System) Severe thermal burns in patients 18 and older Granted clearance by the FDA
Cardurion Pharmaceuticals CRD-733, a Phosphodiesterase-9 (PDE-9) inhibitor Heart failure FDA has accepted Investigational New Drug Application (IND)
OBI Pharma, Inc. OBI-3424 Acute Lymphoblastic Leukemia (ALL) Granted Orphan Drug Designation by FDA
Alnylam Pharmaceuticals Givosiran Acute hepatic porphyrias (AHPs) Successful early Phase III clinical trial results announced
AstraZeneca Imfinzi (durvalumab) Non-small cell lung cancer (NSCLC) Phase III PACIFIC clinical trial found cut deaths by a third
Genentech Tecentriq plus chemotherapy Extensive-stage small cell lung cancer (ES-SCLC) Phase III trial showed treatment helped patients live significantly longer than chemo alone
Daiichi Sankyo Company, Ltd Trastuzumab deruxtecan (DS-8201) Previously-treated HER2 positive unresectable and/or metastatic breast cancer Initiated two Phase III trials
Aurinia Pharmaceuticals, Inc. Voclosporin added to background therapy of mycophenolate mofetil (MMF)/CellCept® Lupus nephritis (LN) Completed enrollment in AURORA Phase III clinical trial
Catabasis Pharmaceuticals, Inc. Edasalonexent Duchenne muscular dystrophy (DMD) Initiated Polaris DMD Phase III clinical trial
AstraZeneca Farxiga (dapagliflozin) Cardiovascular disease Positive Phase III clinical trial results announced
Alexion Pharmaceuticals, Inc. Soliris® (eculizumab) Anti-aquaporin-4 (AQP4) auto antibody-positive neuromyelitis optica spectrum disorder (NMOSD) Positive topline results from the Phase III PREVENT announced

New Pipeline Updates from AstraZeneca, Teva, Camurus and Ironwood

September 24, 2018

Company Drug/Device Medical Condition Status
inui™ Health In-home urine analysis platform, which includes a disposable test and smart phone app (iOS and Android) Kidney function, metabolic disorders and urinary tract infections (UTI) Granted 510(k) clearance and a Clinical Laboratory Improvements Amendments (CLIA) waiver by the FDA CE Mark certification
Camurus CAM2038 Chronic low back pain Met primary and first secondary endpoints; resulted in significantly improved relief of the average and worst pain intensity compared to placebo
Iterum Therapeutics plc Sulopenem Gram-negative, multi-drug resistant infections Initiated SURE 2 and SURE 3 Phase III clinical trials
AstraZeneca Pharmaceuticals LP FASENRA™ (benralizumab) Severe eosinophilic asthma Phase III extension BORA trial results show long-term safety and efficacy as an add-on maintenance treatment
Regeneron Pharmaceuticals, Inc. and Sanofi Dupixent® (dupilumab) mono-therapy moderate-to-severe atopic dermatitis Phase III trial demonstrated a significant improvement in signs and symptoms
Alnylam Pharmaceuticals, Inc. Patisiran Polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults APOLLO Phase III trial showed improved markers of cardiomyopathy
AstraZeneca Triple combination therapy PT010 COPD Seeking FDA approval after Phase III trial found it outperformed its dual-therapy rivals eight of nine times
Teva Pharmaceutical Industries Ajovy (fremanezumab) Migraine Granted approval by the FDA
Venture Heat® Infrared Heat Therapy using Far infrared Rays (FIR) Pain Granted approval by the FDA
Sebacia, Inc. Sebacia Microparticles Mild to moderate inflammatory acne vulgaris Granted clearance by the FDA
WAT Medical HeadaTerm Migraine Granted clearance by the FDA
AstraZeneca Lumoxiti (moxetumomab pasudotox-tdfk) Adults with relapsed or refractory hairy cell leukemia (HCL) who have had at least two previous therapies Granted clearance by the FDA
Ironwood Pharmaceuticals, Inc. Praliciguat (IW-1973) Heart failure Granted Fast Track designation by the FDA
Genentech Subcutaneous (SC) formulation of Actemra® (tocilizumab) Active systemic juvenile idiopathic arthritis (SJIA) in patients two year and up Granted approval by the FDA
STAAR Surgical Company PMA Supplement for the Visian Toric ICL Myopia with astigmatism Granted approval by the FDA

New Pipeline Updates from Amryt, Loxo Oncology, Bristol-Myers Squibb and Pfizer

September 17, 2018

Company Drug/Device Medical Condition Status
Amryt AP101 Epidermolysis Bullosa, a genetic disorder that causes skin and lining of internal organs to blister and tear Granted Investigational New Drug clearance by FDA to open U.S. sites as part of EASE Phase III clinical trial
Fibrocell Science, Inc. FCX-013 Moderate to severe localized scleroderma Granted Fast Track designation by the FDA
Loxo Oncology, Inc. LOXO-292 Metastatic RET-fusion-positive non-small cell lung cancer; RET-mutant medullary thyroid cancer Granted Breakthrough Therapy designation by the FDA
Genesys Spine AIS-C Stand-Alone System a first of its kind, non-screw based, zero-profile, direct-anterior interbody system Cervical spine surgery Granted FDA clearance
Perrigo Company plc OTC equivalent of Imodium® Multi-Symptom Relief (loperamide hydrochloride and simethicone tablets, 2 mg/125 mg) Diarrhea plus bloating, pressure and cramps Granted approval by the FDA
Concentric Analgesics, Inc. CA-008, a non-opioid therapeutic Post-surgical pain Granted Breakthrough Therapy designation by the FDA
physIQ AFib detection analytics engine Atrial Fibrillation Granted FDA 510(k) clearance
Foamix Pharmaceuticals, Ltd. FMX101, tpical minocycline foam Moderate to severe acne Met endpoints in Phase III trial
Merck KGaA and Pfizer Combination of Bavencio (avelumab) and Inlyta (axitinib) Advanced renal cell carcinoma Therapy significantly improved progression-free survival in Phase III trial
Vital Therapies VT-308 Severe alcoholic hepatitis Failed Phase III clinical trial
Gilead Sciences and Galapagos NV Filgotinib Moderate to severe active rheumatoid arthritis FINCH 2 Phase III trial successfully met primary endpoints
Bristol-Myers Squibb BMS-986165 Moderate to severe plaque psoriasis Hit efficacy endpoints in Phase II clinical trial
Amygdala Neurosciences ANS-6637 Alcohol use disorder Initiated Phase II clinical trial with NIAAA
Abeona Therapeutics, Inc. Gene therapy product ABO-101 (AAV-NAGLU) MPS IIIB (Sanfilippo syndrome type B) Initiated Phase I/II clinical trial in Spain
EpicGenetics, Inc. Tuberculosis vaccine, BCG Fibromyalgia Launching Phase II clinical trial with Mass General Hospital
Checkpoint Therapeutics, Inc. CK-101 (also known as RX518), a third-generation epidermal growth factor receptor tyrosine kinase inhibitor (TKI) Non-small cell lung cancer Positive preliminary safety and efficacy data announced from an ongoing Phase I/II clinical trial

New Pipeline Updates from Janssen, Eli Lilly, Gilead and Lysogene

September 10, 2018

Company Drug/Device Medical Condition Status
Janssen Esketamine nasal spray Treatment-resistant depression in adults Submitted a new drug application to the FDA
EpicGenetics BCG (an inexpensive generic vaccine to prevent tuberculosis) Fibromyalgia Massachusetts General Hospital granted FDA approval for human trial
ARMGO Pharma, Inc. ARM210 (also known as S48168) Ryanodine Receptor Type 1 Related Myopathies (RYR1-RM) Granted Orphan Drug Designation by the FDA
Lysogene LYS-SAF302 Mucopolysaccharidosis Type IIIA (MPS IIIA) Granted Investigational New Drug (IND) clearance by FDA for Phase II-III (AAVance) trial in U.S.
Pfizer Oral Janus kinase 3 (JAK3) inhibitor PF-06651600 Alopecia areata, a chronic autoimmune skin disease that causes hair loss Granted Breakthrough Therapy designation by the FDA
ITF Pharma TIGLUTIK™ (riluzole) oral suspension Amyotrophic lateral sclerosis (ALS) Granted approval by the FDA
Baxter International, Inc. Actifuse Flow Bone Graft Substitute Orthopedic surgical procedures Granted clearance by the FDA
AstraZeneca/MedImmune Anifrolumab Moderate to severe lupus erythematosus (SLE) Flunked Phase III clinical trial
Horizon Pharma plc Teprotumumab Moderate-to-severe active thyroid eye disease (TED) Early completion of enrollment for confirmatory Phase III trial
SillaJen/Lee’s Pharmaceutical Holdings Oncolytic immunotherapy Pexa-Vec (formerly JX-594) Advanced liver cancer First patient enrolled in China in the Phase III PHOCUS clinical trial
Eli Lilly and Company Flortaucipir F 18, a Positron Emission Tomography (PET) imaging agent Predicting brain tau pathology and Alzheimer’s disease diagnosis Phase III trial successfully met two primary endpoints
Advicenne ADV7103 Distal Renal Tubular Acidosis (dRTA) Granted Investigational New Drug (IND) clearance by FDA for ARENA-2 Phase III clinical trial
Gilead Sciences and Galapagos NV Filgotinib, a selective JAK1 inhibitor Active ankylosing spondylitis (AS) in adults Phase II TORTUGA clinical trial met endpoint of greater improvements in AS Disease Activity Score (ASDAS)
Neumentum Non-opioid NTM-001 (pre-mixed bag ketorolac for continuous infusion) Moderately severe acute pain Completed dosing the first cohort of patients in its Phase I trial
PvP Biologics, Inc. Kuma062 Celiac disease Two first-in-human clinical trials initiated

New Pipeline Updates from Pfizer, Shire, Poxel and Compass Pathways

September 4, 2018

Company Drug/Device Medical Condition Status
Cycle Pharmaceuticals Ltd Ketorolac Tromethamine Tablets 10mg (as continuation treatment post IV/IM dosing of ketorolac tromethamine) Moderately severe acute pain Granted FDA approval of Abbreviated New Drug Application (“ANDA”)
Compass Pathways Psilocybin, the active ingredient in “magic mushrooms” Treatment-resistant depression Received FDA approval for Phase IIb Clinical Trial
Akcea Therapeutics Waylivra (volanesorsen) Familial chylomicronemia syndrome (FCS) Received Complete Response Letter from FDA
Poxel SA Imeglimin Type 2 diabetes Patient enrollment completed in TIMES 3 trial of the Phase III registration program
Ceterix Orthopaedics, Inc. NovoStitch® Pro Meniscal Repair System – size 0 suture cartridge Meniscal tears 501 (k) clearance granted by the FDA
Reflection Biotechnologies Limited RBIO-101 program (AAV.CYP4V2), an AAV-based gene therapy product Bietti’s Crystalline Dystrophy (BCD) Granted Orphan Drug Designation by the FDA
Berlin Cures BC 007, a DNA-based aptamer compound that binds to and eliminates functional pathogenic autoantibodies Cardiomyopathy Announced successful completion Phase I trial
Novaremed AG Lead compound NRD.E1, a novel, first-in-class, small molecule Diabetic neuropathy pain Announced funding goal reached to begin global Phase IIb trial
Promore Pharma AB LL-37 Venous leg ulcers Approval to start Phase IIb clinical trial in Sweden and Poland
Pfizer Tafamidis Transthyretin Cardiomyopathy Announced Phase III findings that therapy cut death risk by 30 percent in patients with distinct disease type
Ortho Dermatologics New Drug Application for ALTRENO™ (tretinoin 0.05%) lotion Acne vulgaris in patients 9 years of age and older Granted approval by the FDA
Kala Pharmaceuticals, Inc. INVELTYS™ (loteprednol etabonate ophthalmic suspension) 1% Post-operative inflammation and pain following ocular surgery Granted approval by the FDA
Biocodex DIACOMIT (stiripentol) Seizures associated with Dravet syndrome (DS) in patients 2 years of age and older taking clobazam Granted approval by the FDA
Dompé Oervate™ (cenegermin-bkbj ophthalmic solution) neurotrophic keratitis (NK) Granted approval by the FDA
Seqirus Next-generation cell-based vaccine manufacturing process at Holly Springs, N.C. Influenza Granted approval by the FDA
Shire Takhzyro (lanadelumab-flyo) injection Hereditary angioedema (HAE) in people 12 years of age and older Granted approval by the FDA

New Pipeline Updates from Aravive Biologics, Biohaven Pharma and Sun Pharmaceutical

August 27, 2018

Company Drug/Device Medical Condition Status
Aravive Biologics, Inc. AVB-S6-500 Platinum-resistant ovarian cancer Granted Fast Track Designation by the FDA
Biohaven Pharma BHV-0223 Social anxiety and public speaking anxiety disorders Positive data from a proof-of-concept Phase III clinical trial
Sun Pharmaceutical Industries Ltd. Cequa (cyclosporine ophthalmic solution) 0.09 percent Keratoconjunctivitis sicca (dry eye) Approval granted by FDA
Lion TCR Pte. Ltd. LioCyx™ Relapsed liver cancer post-liver transplantation Approval granted by Health Sciences Authority (HSA), Singapore, for multicenter Phase I/II clinical trial
Taiwan Liposome Company, Ltd. TLC590, a proprietary BioSeizer™ liposomal formulation of ropivacaine Postsurgical pain Dosing of first patients in a Phase I/II trial
Teva Pharmaceuticals Generic version of EpiPen Auto-Injector Severe allergic reactions Approval granted by the FDA
BeiGene Ltd. Tislelizumab, an investigational anti-PD-1antibody, combined with chemotherapy as a potential first-line treatment Stage IIIB or IV squamous non-small cell lung cancer (NSCLC) Phase III clinical trial initiated
Eiger BioPharmaceuticals, Inc. Avexitide (formerly exendin 9-39) Post-bariatric surgical patients who experience post-bariatric hypoglycemia (PBH) Completion of enrollment in Phase II clinical trial
Escalier Biosciences, BV ESR-114 topical gel Mild-to-moderate psoriasis Phase I/IIa clinical trial initiated
Sangamo Therapeutics, Inc. SB-525, a cDNA gene therapy candidate Hemophilia A Positive preliminary data from the Phase I/II clinical trial
ADC Therapeutics ADCT-402 (loncastuximab tesirine) Relapsed or refractory diffuse large B-cell lymphoma (DLBCL) Phase II clinical trial initiated
Alexion Pharmaceuticals, Inc. ALXN1210, investigational long-acting C5 complement inhibitor Paroxysmal nocturnal hemoglobinuria (PNH) FDA accepted Biologics License Application (BLA) for approval
Merck & Co. Keytruda in combination with Alimta (pemetrexed) and platinum chemotherapy as first-line treatment Metastatic non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations Granted approval by the FDA
SPR Therapeutics SPRING endura (single lead) and extensa (dual lead) Peripheral Nerve Stimulation (PNS) Systems Pain Cleared by the FDA
Univision 360 Luminelle DTx Hysteroscopy System Hysteroscopy and cystoscopy Received 510(k) clearance from the FDA
Tolero Pharmaceuticals, Inc. Combination therapy with AbbVie’s venetoclax and Tolero’s investigational agent Relapsed/refractory acute myeloid leukemia (AML) Tolero Pharmaceuticals and AbbVie partner for trial

New Pipeline from Ivantis, Genentech, BeiGene and Endomag

August 20, 2018

Company Drug/Device Medical Condition Status
Amicus Therapeutics Galafold (migalastat) Fabry disease Approval granted by the FDA
Arbutus Biopharma Coporation and Alnylam Pharmaceuticals, Inc. ONPATTRO (patisiran) Hereditary transthyretin amyloidosis Approval granted by the FDA
Ivantis, Inc. Hydrus® Microstent, a microinvasive glaucoma surgery (MIGS) device Mild to moderate primary open-angle glaucoma in conjunction with cataract surgery Approval granted by FDA
Genentech Xlair® (omalizumab) Prevention of severe allergic reactions following accidental exposure to foods in people with allergies Breakthrough Therapy Designation granted by the FDA
Samsung Bioepis, Co., Ltd. SB26 ulinastatin-Fc (UTI-Fc) fusion protein Severe acute pancreatitis Phase I trial set to begin
Sesen Bio, Inc. Vicinium™ Non-muscle invasive bladder cancer (NMIBC) Granted Fast Track Designation by the FDA
BeiGene Ltd. Tislelizumab, an investigational anti-PD-1 antibody, combined with chemotherapy Potential first-line treatment for patients with Stage IIIB or IV squamous non-small cell lung
cancer (NSCLC)
Phase III clinical trial initiated
Eiger BioPharmaceuticals, Inc. Avexitide (formerly exendin 9-39) Post-bariatric surgical patients who experience post-bariatric hypoglycemia (PBH) Completion of enrollment in Phase II clinical trial
Izana Bioscience Namilumab Ankylosing spondylitis Phase II proof-of-concept clinical trial initiated
Rafael Pharmaceuticals, Inc. CPI-613 in combination with bendamustine Relapsed or refractory T-Cell Lymphoma Activation of a second clinical trial site for the Phase I study
Endomag Magtrace, the first non-radioactive, dual-tracer Lymphatic mapping in patients with breast cancer undergoing a mastectomy Granted premarket approval granted by the FDA
Cannabics Pharmaceuticals, Inc. Cannabics SR 5mg, daily for 3 months Advanced Cancer and Cancer Anorexia Cachexia Syndrome (CACS) Conclusion of trial announced; officials results are being evaluated
OrthoTrophix, Inc. TPX-100 Osteoarthritis Phase II trial results: improvements in function and cartilage in 55 percent of TPX-100 treated knees
Entasis Therapeutics ETX2514SUL Complicated urinary tract infections, including acute pyelonephritis (kidney infection) in adults Positive topline results from Phase II trial
Realm Therapeutics PR022 Atopic dermatitis Phase II trial failed to reach endpoint goals
INOVA Diagnostics, Inc. QUANTA Flash® HMGCR Aids in the diagnosis of idiopathic inflammatory myopathy (IIM), a group of conditions that affect the skeletal muscles Received FDA clearance

New Pipeline from BioCryst, Elite, PharmaMar and Vertex

August 13, 2018

Company Drug/Device Medical Condition Status
OncoPep, Inc. PVX-410, investigational multi-peptide cancer vaccine Smoldering multiple myeloma (SMM) Phase Ib trial initiated
SW Safety Solutions, Inc. Nitrile Exam Gloves with Low Dermatitis Potential Claim Skin irritation or sensitization Initial – Looking for Principal Investigator to head clinical study
Kyowa Kirin, Inc. Poteligo (mogamulizumabkpkc) injection Relapsed or refractory mycosis fungoides (MF) or Sezary syndrome after at least one prior systemic therapy Approval granted by FDA
Celltrion, Inc. Bevacizumab biosimilar ‘CT-P16’ Metastatic colorectal cancer, metastatic breast cancer, non-small cell lung cancer, and glioblastoma Phase III trial initiated
Apotex Inc. New generic potassium chloride oral solution Hypokalemia (low potassium blood levels) in patients who are on diuretics Approval granted by the FDA under a novel term called the Competitive Generic Therapy designation
Alector, LLC AL001, a human recombinant monoclonal antibody Frontotemporal dementia (FTD) Granted Orphan Drug Designation by the FDA
Pliant Therapeutics, Inc. PLN-74809 Idiopathic pulmonary fibrosis (IPF) Granted Orphan Drug designation by the FDA
Castle Creek Pharmaceuticals, LLC Diacerein 1% ointment (CCP-020) Epidermolysis bullosa simplex (EBS) Fast Track Designation granted by the FDA
Elite Pharmaceuticals, Inc. Generic methadone hydrochloride 5 mg and 10 mg tablets Severe pain, maintenance treatment of opioid addiction (heroin or other morphine-like drugs) Approval granted by the FDA
Alteogen Inc. ALT-P7, an antibody-drug conjugate (ADC) using a Trastuzumab variant form of antibody Gastric cancer Granted Orphan Drug Designation by the FDA
Vertex Pharmaceuticals Inc. Orkambi (lumacaftor/ ivacaftor) Underlying cause of cystic fibrosis in children ages 2 to 5 with the most common form of the disease Approval granted by the FDA
BioCryst Pharmaceuticals, Inc. BCX7353 Prevention of angioedema attacks in patients with hereditary angioedema Fast Track Designation granted by the FDA
PharmaMar Lurbinectedin Small cell lung cancer Granted Orphan Drug Designation by the FDA
Sensus Healthcare, Inc. SRT-100+, the next-generation Superficial Radiation Therapy solution Non-melanoma skin cancers (NMSC) and keloids Granted 501(k) clearance by the FDA
Armagen Inc. GT-184, an investigational enzyme replacement therapy (ERT) Mucopolysaccharidosis type IIIA (also known as Sanfilippo Syndrome A or MPS IIIA) Granted Orphan Drug Designation by the FDA
Shionogi & Co., Ltd Mulpleta®(lusutrom-bopag) Thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo a procedure Approval granted by the FDA