Envarsus XR sNDA Seeking New Indication Submitted to FDA

By March 08, 2018

In the study, Envarsus XR proved non-inferior to immediate-release tacrolimus as seen by treatment failure rates.

Lemborexant, Zolpidem ER Go Head-to-Head in Sleep Disorder Study

By March 07, 2018

In the Phase 3 SUNRISE 1 study, lemborexant achieved its primary and key secondary objectives vs. placebo and vs. zolpidem tartrate extended-release (active comparator) in patients aged ≥55 years with difficulty staying asleep through the night.

FDA Accepts NDA for Novel Chronic Idiopathic Constipation Treatment

By March 05, 2018

If approved, prucalopride will be the only readily available 5-HT4 agonist for adult CIC in the U.S.

Dupilumab Under FDA Review for Moderate-to-Severe Asthma

By March 02, 2018

Dupixent is a human monoclonal antibody specifically designed to inhibit signaling of interleukin-4 (IL-4) and interleukin-13 (IL-13), two important signaling proteins (cytokines) that contribute to Type 2 inflammation in moderate-to-severe asthma.

FDA Accepts Solriamfetol NDA for Narcolepsy-, OSA-Related Sleepiness

By March 02, 2018

Solriamfetol is a selective dopamine and norepinephrine reuptake inhibitor.

First-in-Class Pancreatic Cancer Candidate Gets Fast Track Status

By March 01, 2018

Pamrevlumab is a first-in-class antibody developed by FibroGen to inhibit the activity of connective tissue growth factor.

FDA to Review NDA for Opioid Analgesic Remoxy ER

By March 01, 2018

Remoxy ER is an abuse-deterrent, extended-release capsule formulation of oxycodone.

BLA for Extended Half-Life Factor VIII Hemophilia A Tx Filed With FDA

By February 27, 2018

N8-GP (turoctocog alfa pegol) is a glycopegylated form of turoctocog alfa designed for prolonged half-life.

Lusutrombopag Under Priority Review for Thrombocytopenia in Liver Disease

By February 26, 2018

The primary endpoint was the number of patients who required no platelet transfusion priory to primary invasive procedure and no rescue therapy for bleeding for 7 days post-procedure.

Pharmacologic Tx for Severe Neonatal Jaundice to Be Reviewed by FDA

By February 23, 2018

The FDA has set an action date of August 22, 2018 to respond to the NDA

Peanut Allergy Oral Immunotherapy Effective in Phase 3 Study

By February 21, 2018

With regard to adverse events, 12.4% of AR101-treated patients discontinued treatment due to treatment-related side effects; these included gastrointestinal adverse events (6.7%) and systemic allergic hypersensitivity reactions (2.7%).

FDA Accepts NDA for Orally Inhaled Levodopa Therapy Inbrija

By February 21, 2018

Inbrija (previously known as CVT-301) is a self-administered, orally inhaled levodopa; it is designed to deliver a precise dose of a dry powder formulation of L-dopa to the lung.

Ready-to-Use Rescue Pen for Severe Hypoglycemia Moves Closer to Approval

By February 16, 2018

The full findings of the trials will not be released until later this year however the Company stated that the trials were a success, with the data already shared with the FDA in a pre-New Drug Application (NDA) meeting.

Rituxan Granted Priority Review for Rare Autoimmune Disease

By February 16, 2018

The disease can be life threatening. Phase 3 study results released last March demonstrated Rituxan substantialy improved pemphigus vulgaris remission rates and successful tapering.

Oral JAK1 Inhibitor Gets Breakthrough Tx Designation for Atopic Dermatitis

By February 15, 2018

In December, a Phase 3, randomized, double-blind, placebo-controlled trial (JADE) was initiated to evaluate the safety and efficacy of PF-04965842 in 375 patients aged ≥12 years with moderate to severe AD.

Selumetinib Gets Orphan Drug Designation for Neurofibromatosis Type 1

February 15, 2018

NF1 has no known cure and is associated with highly-variable symptoms

FDA Accepts NDA for Plaque Psoriasis Treatment Jemdel

By February 14, 2018

Jemdel, if approved, will be the first high-potency topical steroid for plaque psoriasis with dosing for 8 weeks. The most common adverse event in clinical trials of Jemdel was upper respiratory tract infection.

Study Evaluating Verubecestat in Prodromal Alzheimer's Disease Stopped

By February 14, 2018

APECS is a randomized, placebo-controlled, parallel-group, double-blind Phase 3 clinical trial evaluating the efficacy and safety of verubecestat in people with prodromal AD.

Positive Results for Ixekizumab in the Treatment of Ankylosing Spondylitis

By February 13, 2018

Taltz demonstrated a statistically significant improvement in the signs and symptoms of AS.

Novel Therapy for Major Depressive Disorder Gets Breakthrough Designation

By February 08, 2018

The Breakthrough Therapy designation is intended to offer a potentially expedited development path and review for promising drug candidates.

Treatment for Smallpox Gets FDA Priority Review

By February 07, 2018

The treatment was developed under the FDA's 'Animal Rule'; where efficacy is determined in animal studies and human studies determine safety and appropriate dosing. The FDA have stated a target action date for TPOXX of August 8, 2018.

Nymox Plans to Submit NDA for Novel Benign Prostatic Hyperplasia Treatment

By February 07, 2018

Fexapotide triflutate 2.5 mg was found to be safe and effective for long-term treatment of BPH.

Positive Results for Ubrogepant in Phase 3 Migraine Trial

By February 06, 2018

A total of 1,327 migraine patients were treated in the study for a single migraine attack. At 2 hours after migraine attack, 19.2% (n=423) and 21.2% (n=448) of ubrogepant 50mg and 100mg were pain free, respectively.

'Gene Silencing' Drug Under FDA Review for Hereditary ATTR Amyloidosis

By February 02, 2018

Patisiran has been granted Fast Track Designation, Breakthrough Therapy Designation, and an expanded Orphan Drug Designation for ATTR amyloidosis from the FDA.

Apomorphine Film Effectively Converts OFF to ON State in PD Patients

By January 30, 2018

The difference in score change for movement disorder from baseline to 30 minutes post-dose between the apomorphine and placebo group was 7.6 (P=0.0002).

Intranasal Acromegaly Treatment Gains Orphan Drug Designation

By January 30, 2018

Octreotide is a somatostatin analog that suppresses excessive growth hormone production from pituitary adenomas.

Extended-Release Oxycodone Prodrug Gets Fast-Tracked

By January 30, 2018

A Phase 1 trial of PF614 compared the treatment to Oxycontin in healthy subjects and found that it demonstrated safety and extended-release characteristics of the prodrug delivery.

Zika Vaccine Candidate Gets Fast Track Designation

By January 29, 2018

A total of 240 individuals between the ages 18 and 49 are enrolled in the Phase 1 trial of TAK-426.

Erenumab Effective in Migraine Patients With Multiple Treatment Failures

By January 23, 2018

Aimovig is a fully human monoclonal antibody, designed to selectively block the calcitonin gene-related peptide (CGRP) receptor.

Trans-Capsaicin Injection Gets Fast Track Status for Knee OA Pain

By January 16, 2018

CNTX-4975 works by targeting the capsaicin receptor (TRPV1) to selectively and rapidly inactivate the local pain fibers transmitting signals to the brain.

Potential First-in-Class Treatment Fast-Tracked for Alopecia Areata

By January 12, 2018

Currently, there are no FDA approved treatments for alopecia areata.

NDA Submitted for Durasert 3-Year Implant for Posterior Segment Uveitis

By January 08, 2018

The NDA submission was supported by data from two Phase 3 studies that met the primary efficacy endpoint of prevention of recurrence of uveitis at 6 months of follow-up (P<0.001).

Significant Migraine Risk Reduction Seen With Eptinezumab in Phase 3 Trial

By January 08, 2018

The 'PROMISE 2' study included 1,072 patients who were randomized to receive eptinezumab (300mg or 100mg), or placebo once every 12 weeks.

FDA to Review New Treatment for Post-Op Ocular Pain, Inflammation

By January 08, 2018

If approved Inveltys would become the first twice-daily post-surgical topical treatment; all current treatments are approved for four-times-a-day dosing.

Plazomicin Gets Priority Review for Complicated UTI, Bloodstream Infections

By January 03, 2018

A 2016 Phase 3 trial (EPIC) including 609 patients with cUTI and acute pyelonephritis found plazomicin to be non-inferior to meropenum.

Eravacycline NDA Submitted for Complicated Intra-Abdominal Infections

By January 03, 2018

Eravacycline is a novel, fully-synthetic fluorocycline antibiotic being developed for the treatment of serious infections, including those caused by multidrug-resistant (MDR) pathogens that have been highlighted as urgent public health threats by both the World Health Organization and the U.S. Centers for Disease Control & Prevention (CDC).

Azedra Under FDA Review for Rare Neuroendocrine Tumors

By January 02, 2018

Azedra, a high-specific-activity radiotherapeutic product, is a substrate for the norepinephrine reuptake transporter that is highly expressed on the cell surface of neuroendocrine tumors.

Epidiolex Under Review for Two Childhood-Onset Epilepsy Conditions

By December 28, 2017

Epidiolex is a pharmaceutical formulation of purified cannabidiol

Cannabidiol Oral Solution Fast Tracked for Rare Pediatric Disorder

By December 27, 2017

The study's primary objectives will be to assess the efficacy of cannabidiol oral solution on hyperphagia-related behavior and body weight in patients with Prader-Willi syndrome

Novel Therapy Looks Promising for Treating Antibiotic-Resistant Bacteria

By December 26, 2017

The patient-the first to be treated with human polyclonal IgG harvested from Tc bovine-received infusions to combat the infection.

FDA to Review Seysara for the Treatment of Moderate to Severe Acne

By December 26, 2017

Seysara (sarecycline) is a once-daily, oral, narrow spectrum tetracycline-derived antibiotic.

Treatment for Angelman Syndrome Gets Fast Track Status

By December 19, 2017

Angelman syndrome affects approximately 1 in 12,000 to 20,000 in the U.S. and symptoms include delayed development, intellectual disability, and seizures.

Positive Results Announced for Cariprazine in Bipolar Depression

By December 19, 2017

Results found that both groups had significantly greater improvements than placebo for the change from baseline to week 6 (P<0.05).

FDA to Review Fremanezumab for Migraine Prevention

By December 19, 2017

Results from the CM trial found that the fremanezumab arm experienced statistically significant reduction in the number of monthly headache days of at least moderate severity vs. placebo.

Fingolimod Gets Breakthrough Therapy Designation for Pediatric MS

By December 18, 2017

The FDA has granted Breakthrough Therapy designation for fingolimod for the treatment of children and adolescents 10 years of age or older with relapsing multiple sclerosis (MS).

Treatment for Leber's Hereditary Optic Neuropathy Gains Fast Track Status

By December 18, 2017

Elamipretide has also been granted Fast Track designation for treating primary mitochondrial myopathy (PMM) - which gained Orphan Drug Designation in September - and Barth syndrome

FDA Lifts Hold on Potential Hemophilia Treatment

By December 15, 2017

Fitusiran, an RNAi therapeutic targeting antithrombin (AT), is designed to lower levels of AT in order to further sufficient thrombin generation to restore hemostasis and prevent bleeding.

Xarelto sNDA Submitted for Two New Vascular Indications

By December 11, 2017

For this group the risk of stroke was reduced by 42% (HR 0.58; 95% CI, 0.44-0.76; P<0.001), CV death by 22% (HR 0.78; 95% CI, 0.64-0.96; P=0.02) and heart attack by 14% (HR 0.86; 95% CI, 0.70-1.05; P=0.14).

FDA to Review Galcanezumab for Migraine Prevention in Adults

By December 11, 2017

The application includes data from three Phase 3 trials; Evolve-1, Evolve-2 (6 months) and Regain (3 months).

Sanofi Discontinues C. Difficile Tx Development

By December 08, 2017

Data that has been collected from the trial so far will be analyzed and shared with the scientific community in due time.

Early Treatment With Kalydeco Investigated in Young Cystic Fibrosis Patients

By December 08, 2017

Sweat chloride, a diagnostic characteristic of CF, was also considerably reduced with ivacaftor treatment. Median baseline sweat chloride levels were 104.1mmol/L.

Novel Post-Surgical Pain Drug Granted Fast Track Designation

By December 07, 2017

An ongoing Phase 1b trial is currently testing CA-008 in patients undergoing bunionectomy, with results expected in the first quarter of 2018.

Congenital Adrenal Hyperplasia Treatment Granted Orphan Drug Status

By December 07, 2017

CAH is a rare endocrine disorder caused by genetic mutations which result in an inability to produce cortisol.

FDA to Review Novel Buprenorphine SL Spray for Acute Pain

By December 06, 2017

Compared to other commonly used opioids, buprenorphine offers a ceiling effect for respiratory depression and less abuse potential, less cognitive impairment, and less constipation.

Pediatric Ulcerative Colitis Tx Granted Orphan Drug Status

By December 04, 2017

The designation is based on a review of Phase 1b clinical data which showed potential for SER-287 as a novel treatment modality for patients with UC

Phase 3 Results Show Further Efficacy of Novel Plaque Psoriasis Tx

By December 04, 2017

At 16 weeks, 73% of risankizumab patients achieved PSAI 90 vs. 2% of the placebo patients and sPGA 0/1 was reached by 84% of riskanizumab patients vs. 7% of placebo patients (P<0.001).

Novel Therapy Shown to Significantly Cut Monthly Migraine Days

By November 30, 2017

Migraine days were cut by ≥50% for half of the 140mg group, by 43.3% for the 70mg group, and by 26.6% for the placebo group.

NDA Under Priority Review for Thrombocytopenia in Chronic Liver Disease

By November 28, 2017

A Prescription Drug User Free Act (PDUFA) date has been set for May 21, 2018.

Hemophilic Arthropathy Treatment Gets Orphan Drug Designation

By November 27, 2017

TRM-201 (rofecoxib) is a highly potent COX-2 selective non-steroidal anti-inflammatory drug (NSAID) with a well-established efficacy profile.

FDA Grants Priority Review to Lofexidine for Opioid Withdrawal Management

By November 21, 2017

Lofexidine, an oral tablet, is a selective alpha 2-adrenergic receptor agonist that reduces the release of norepinephrine.

Cervical Dystonia Drug Candidate Gets Orphan Drug Designation

By November 21, 2017

Treatments for cervical dystonia include oral medications, botulinum toxin injections, surgery, and complementary therapies.

FDA Grants Fast Track Status to Lumateperone for Schizophrenia

By November 20, 2017

In two studies, lumateperone showed a statistically significant separation from placebo on the primary endpoint, the Positive and Negative Syndrome Scale, or PANSS, total score.

Patidegib Gets Breakthrough and Orphan Designation for Gorlin Syndrome

By November 20, 2017

Gorlin Syndrome is a rare, genetic disease characterized by mutations in the tumor suppressor gene encoding Patched1.

Agranulocytosis Cases Prompt More Monitoring for Investigational Parkinson's Drug

By November 16, 2017

Dependent on further input from the Data Safety Monitoring Board (DSMB) and the Food and Drug Administration (FDA), the Company stated that they intend on moving forward with the study and expect to report results in the first quarter of 2018.

NDA Submitted for Revefenacin for the Treatment of COPD

By November 14, 2017

In the Phase 3 trials revefenacine demonstrated statistically significant and clinically meaningful improvements compared to placebo in trough forced expiratory volume in 1 second (FEV1).

Positive Results for Brexanolone in Postpartum Depression Trials

By November 10, 2017

The Hummingbird Phase 3 program included two Phase 3, multicenter, randomized, double-blind, parallel-group, placebo-controlled trials designed to evaluate the safety and effectiveness of brexanolone in women with moderate and severe PPD.

Novel Malaria Drug Gets Orphan Drug Designation

By November 07, 2017

s well as malaria, they are also evaluating Artemisone for the treatment of human cytomegalovirus (CMV) infections, including stem cell transplant CMV and congenital CMV.

NDA Submitted for Olinvo to Treat Moderate to Severe Acute Pain

By November 02, 2017

Olinvo is a next generation IV analgesic for the management of moderate-to-severe acute pain in the hospital and similar settings and has been granted Breakthrough Therapy designation by the FDA.

FDA Accepts NDA for Topical Combination Treatment for Plaque Psoriasis

By November 02, 2017

The Prescription Drug User Fee Act (PDUFA) action date is June 18, 2018.

Long-Term Efficacy Data Show Sustained Pelvic Pain Reduction With Elagolix

By November 01, 2017

No new safety concerns were identified with elagolix use for the 12-month treatment period

Novel Monoclonal Antibody Targeting Influenza A Gets Fast Track Status

By November 01, 2017

VIS410, a monoclonal antibody, is being developed for single-dose administration to target all known strains of influenza A.

Aemcolo Gets QIDP, Fast Track Designation for Travelers' Diarrhea

By October 31, 2017

In Phase 3 clinical trials involving patients with travelers' diarrhea, Aemcolo was found to be superior to placebo (P=0.0008) and non-inferior to ciprofloxacin.

Dupilumab Beneficial for Patients with Severe Steroid-Dependent Asthma

By October 31, 2017

Consistent reductions in asthma attacks and improvements in lung function across a broad population of uncontrolled asthma patients.

Novartis Seeks New Indication for CAR-T Therapy Kymriah

By October 31, 2017

The latest sBLA is intended for patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) who are ineligible for autologous stem cell transplant (ASCT).

BLA for Investigational Hemophilia A Treatment Accepted

By October 31, 2017

BAY94-9027 is designed to prolong FVIII activity in the blood while preserving coagulation activity using site-specific technology in which the Polyethylene glycol molecule is consistently attached to the factor VIII protein at a specific site.

Influenza Oral Tablet Vaccine Shows Promise in Phase 2 Trial

By October 27, 2017

Results showed that the tablet group had a 39% reduction in clinical disease compared to placebo and a 27% reduction compared to the injectable QIV.

Opioid Alternative Tx for Surgical Site Granted Fast Track Status

By October 26, 2017

In bunionectomy and hernia repair HTX-011 (60mg and 300mg, respectively) was found to significantly reduced pain through 72 hours compared to placebo and bupivacaine (P=0.0003 and P=0.0106).

Orphan Drug Status Granted to Tx for Rare Bowel Disorder

By October 25, 2017

A Phase 2 trial demonstrated how glepaglutide treatment was associated with reduced fecal wet weight output as well as indicated increases in energy, fluid and electrolyte absorption.

FDA Grants Alzheimer's Disease Drug Candidate Fast Track Status

By October 25, 2017

The company said in a press release that their pivotal Phase 3 program for ALZ-801 will focus on approval in the genetically-defined subpopulation of high risk patients who are at the Mild stage of AD.

Valbenazine Granted Orphan Drug Designation for Tourette Syndrome

By October 25, 2017

Valbenazine is already approved and marketed as Ingrezza in the US to treat adults with tardive dyskinesia

Disease-Modifying Osteoarthritis Tx Granted Fast Track Status

By October 25, 2017

There are currently no DMOADs approved for use in OA. MIV-711 is a selective inhibitor of cathepsin K, which is the principal protease involved in breaking down collagen in bones and cartilage.

Treatment for Fistulizing Crohn's Disease Gets Orphan Drug Status

By October 24, 2017

Cx601 is a first-in-class allogeneic cell therapy intended for patients who have had an inadequate response to at least one conventional or biologic therapy.

First-in-Class, Oral Agent Gets Orphan Drug Status for Pancreatic Cancer

By October 20, 2017

Mesupron is a first-in-class, orally-administered protease inhibitor.

BLA Submitted for Migraine Prevention Drug Fremanezumab

By October 17, 2017

The studies looked at the safety, tolerability, and efficacy of 4 dose regimens of subcutaneous fremanezumab vs. placebo over 16 weeks.

First-in-Class Alzheimer's Drug Gets Fast-Tracked by FDA

By October 16, 2017

CT1812 is a first-in-class, orally-administered small molecule that targets the sigma-2 receptor complex on neuronal synapses, displacing beta amyloid oligomers from their binding sites on brain cells and clearing them in to the cerebrospinal fluid.

Investigational Hyperhidrosis Therapy Found to Be Safe, Effective

By October 13, 2017

Daily treatment with glycopyrronium tosylate in the Phase 3, open-label ARIDO trial was generally well-tolerated during 44 weeks of treatment.

First Gene Tx for Inherited Retinal Disease Gets Thumbs Up from FDA Committee

By October 13, 2017

The recommendation was based on data from the Luxturna clinical development program which included a Phase 3 trial in patients 4-44 years of age.

Merck Decides to Put the Brakes on CETP Inhibitor Anacetrapib

By October 13, 2017

The decision follows a review of the clinical profile and discussions with external experts

Combo Antibiotic Gets Priority Review for New Pneumonia Indication

By October 13, 2017

Avycaz is a combination antibiotic consisting of ceftazidime, a cephalosporin, and avibactam, a beta-lactamase inhibitor.

Leprosy Vaccine Candidate to Be Tested in Humans

By October 12, 2017

Leprosy (also known as Hansen's disease) is caused by the Mycobacterium leprae bacteria and results in disfiguration of the skin and mucous membranes as well as incurable nerve damage.

NDA Submitted for Novel Non-Metastatic CRPC Treatment

By October 11, 2017

Apalutamide, an investigational, next generation oral androgen receptor inhibitor, was assessed for safety and efficacy in the Phase 3 pivotal trial, ARN-509-003 (SPARTAN), which the NDA is based on.

PLK1 Inhibitor Granted Orphan Drug Designation for AML

By October 10, 2017

A Phase 1b/2 open-label study is being planned to assess the safety and efficacy of PCM-075.

First-in-Class Oral Tx for Uterine Fibroids One Step Closer to Approval

By October 10, 2017

Ulipristal acetate is a selective progesterone receptor modulator which acts directly on the progesterone receptors in the endometrium, uterine fibroids, and the pituitary gland.

Lixivaptan Gets Orphan Drug Status for Autosomal Dominant PKD

By October 05, 2017

Lixivaptan is a potent, selective vasopressin V2 receptor antagonist which has been found to delay the progression of an autosomal dominant form of polycystic kidney disease.

Merck Discontinues Clinical Program for Two HCV Treatments

By September 29, 2017

The MK-3682B trial evaluated a triple-combination therapy.

Novel Buprenorphine Sublingual Spray NDA Submitted

By September 29, 2017

Buprenorphine is a partial agonist of the mu-receptor and an antagonist of the kappa receptor, which contributes to its analgesic effects.

FDA: More Data Needed for Sirukumab for Active RA

By September 27, 2017

The CRL indicated a request for additional clinical data in order to evaluate the safety of sirukumab in the treatment of RA.

No Significant Improvement with Intepirdine in Alzheimer's Disease Study

By September 26, 2017

The study included patients with mild to moderate Alzheimer's disease on donepezil therapy (n= 1315) and compared once-daily oral doses of intepirdine 35mg to placebo over 24 weeks.

Linhaliq NDA Granted Priority Review

By September 25, 2017

Linhaliq was previously granted Qualified Infectious Disease Product (QIDP) Designation and Fast Track designation for NCFBE with lung infections with P. aeruginosa.

Pompe Disease Treatment Granted Orphan Drug Designation

By September 25, 2017

ATB200 is a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures while AT2221 is a pharmacological chaperone.