Familial Chylomicronemia Syndrome Treatment to Get FDA Review

By August 31, 2017

A total of 66 patients with FCS were enrolled in the year-long APPROACH which met its primary endpoint of reduction in triglycerides at 3 months, with a 77% mean reduction in triglycerides for volanesorsen-treated patients.

FDA Greenlights MDMA Studies for Posttraumatic Stress Disorder

By August 31, 2017

"Our Phase 2 data was extremely promising with a large effect size, and we are ready to move forward quickly," said Amy Emerson, Executive Director of the MAPS Public Benefit Corporation.

FDA to Review Novel ADHD Treatment Dasotraline

By August 31, 2017

Two year-long studies assessing safety of dasotraline in children, adolescent and adult ADHD patients found the treatment to be generally well tolerated.

Tezacaftor/Ivacaftor Gets Priority Review for Cystic Fibrosis

By August 25, 2017

The FDA has granted Priority Review to the New Drug Application (NDA) of tezacaftor/ivacaftor (Vertex) for the treatment of patients ≥12yrs old with cystic fibrosis (CF) who have two copies of the F508del mutation or one F508del mutation and one residual function mutation.

BLA for Novel Hemophilia A Therapy Granted Priority Review

By August 24, 2017

The FDA has accepted and granted Priority Review to the Biologics License Application (BLA) of emicizumab (Genentech) for the prophylactic treatment of hemophilia A in adults, adolescents and children with factor VIII inhibitors.

BLA Submitted for Novel X-Linked Hypophosphatemia Tx

By August 24, 2017

Burosumab is being developed in a collaboration between Ultragenyx and Kyowa Hakko Kirin Pharmaceuticals, based on a license agreement between both companies.

Adcetris sBLA Granted Priority Review for Cutaneous T-Cell Lymphoma

By August 17, 2017

Key secondary endpoints included complete response rate, progression-free survival (PFS), and reduction in the burden of symptoms during treatment.

Novel Reversal Agent for Factor Xa Inhibitors Under FDA Review

By August 17, 2017

The FDA has accepted for review the BLA resubmission for AndexXa (andexanet alfa; Portola) for the reversal of the anticoagulant effects of Factor Xa inhibitors in patients experiencing uncontrolled or life-threatening bleeding.

First-in-Class Dual SGLT1/2 Inhibitor Shows Sustained HbA1c Reduction

By August 16, 2017

The overall mean placebo-adjusted A1C reduction at week 24 was 0.37% in the 200mg dose arm (P<0.001) and 0.35% in the 400mg dose arm (P<0.001), this was sustained over the 52-week duration of the study.

Fixed-Dose, Single-Tablet HIV Regimen Granted Priority Review

By August 14, 2017

The investigational fixed-dose treatment combines bictegravir (BIC) 50mg, a novel integrase strand transfer inhibitor (INSTI), with emtricitabine (FTC) 200mg and tenofovir alafenamide (TAF) 25mg, a dual-NRTI backbone.

Regeneron to Discontinue Development of RSV Treatment

By August 14, 2017

The primary endpoint of RSV prevention - assessed through day 150 of treatment - was not significantly reached.

NDA Submitted for Non-Cystic Fibrosis Bronchiectasis Treatment

By August 11, 2017

Linhaliq (previously known as 'Pulmaquin') consists of a mixture of liposome encapsulated and unencapsulated ciprofloxacin. Linhaliq has previously been granted Orphan Drug status for the management of bronchiectasis.

Novel lgA Nephropathy Tx Gains Orphan Drug Status

By August 11, 2017

Phase 2 trials have shown to be associated with reductions in urine protein levels following treatment with OMS721 in patients with lgA nephropathy.

FDA Grants Zelboraf Priority Review for Rare Blood Disease

By August 07, 2017

The application includes data from the open-label, Phase 2 VE-BASKET study; non-randomized, basket study investigating the use of Zelboraf in patients with BRAF V600 mutation-positive cancers and other diseases, including 22 patients with ECD.

Mytesi Granted Orphan Drug Designation for Short Bowel Syndrome

By August 07, 2017

Clinical studies of Mytesi have shown that the most common adverse reactions were upper respiratory tract infection (5.7%), bronchitis (3.9%), cough, and flatulence (3.1%).

Tesevatinib Gains Orphan Drug Status for EGFR-Mutation Positive NSCLC

By August 07, 2017

In March 2016, tesevatinib was designated Orphan Drug status for the treatment of autosomal recessive polycystic kidney disease (ARPKD).

Investigational PNH Treatment Granted Orphan Drug Status

By August 04, 2017

"We have designed RA101495 for once-daily, subcutaneous self-administration, an approach which has the potential to ease this burden, improve convenience, and provide much-needed dosing flexibility," said Doug Treco, PhD, President and CEO of Ra Pharma.

Investigational PNH Treatment Granted Orphan Drug Status

By August 04, 2017

"We have designed RA101495 for once-daily, subcutaneous self-administration, an approach which has the potential to ease this burden, improve convenience, and provide much-needed dosing flexibility," said Doug Treco, PhD, President and CEO of Ra Pharma.

Investigational Migraine Drug Lasmiditan Effective in Phase 3 Study

By August 04, 2017

Results showed that a statistically significant number of those participants who received lasmiditan (in all doses) were pain-free at 2-hours post dosage compared to those who received placebo (the primary endpoint).

Obeticholic Acid + Statin Shows Rapid Reversal of LDL Rise in NASH Patients

By August 02, 2017

The mean LDL change from baseline to Week 16 were as follows: placebo: -48 mg/dL, OCA 5mg: -40mg/dL, OCA 10mg: -40mg/dL, and OCA 25mg: -45mg/dL.

Imfinzi Designated Breakthrough Therapy for NSCLC

By July 31, 2017

Imfinzi, an anti-PD-L1 monoclonal antibody, was previously granted accelerated approval from the FDA for locally advanced or metastatic urothelial carcinoma in patients who have disease progression during or after platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant platinum-containing chemotherapy.

Repatha sBLA Under Priority Review for MACE Risk Reduction

By July 28, 2017

Results showed that the evolocumab group had a statistically significant 15% reduction (P<0.001) in the risk of extended MACE composite (primary) endpoint - which included hospitalization for unstable angina, coronary revascularization, heart attack, stroke or cardiovascular death - compared to placebo group.

Epinephrine Auto-Injector for Infants, Small Children Gets Priority Review

By July 27, 2017

The company has indicated that if the new auto-injector is approved they expect it to be available for patients in the first half of 2018.

Caplacizumab Fast-Tracked for Acquired Thrombotic Thrombocytopenic Purpura

By July 26, 2017

The Phase 2 TITAN study (n=75) evaluated the safety and efficacy of caplacizumab with standard of care of plasma exchange and immunosuppression.

Vaccine for Recurrent UTI Granted Fast Track Designation

By July 26, 2017

The vaccine consists of the FimH bacterial adhesion protein as well as a new adjuvant designed to induce an immune response to prevent bacterial colonization of the urinary tract.

Investigational Antibiotic Combo Beats Best Available Tx for CRE

By July 26, 2017

Patient randomization was discontinued early after an independent Data and Safety Monitoring Board (DSMB) determined that a risk-benefit analysis (n=72) of available data no longer necessitated additional patient randomization to the comparator arm (best available therapy).

ALS-Related Muscle Cramp Drug Gains Fast Track Status

By July 25, 2017

Two Phase 2 trials of FLX-787 titled COMMAND and COMMIT are expected to start in the U.S. this quarter in ALS and Charcot-Marie-Tooth (CMT) patient populations, respectively.

Lyme Disease Vaccine Gets Fast Track Status

By July 24, 2017

Additionally, the study will measure immunogenicity against the 6 most prevalent serotypes of Lyme borreliosis present in the vaccine.

Investigational Nebulized LAMA Shows Promise in COPD Trial

By July 19, 2017

Results found that rates of COPD exacerbations were lowest in the 175mcg treatment arm. Also, the adverse events and serious adverse events in the revefenacin group were comparable to the tiotropium group.

FDA Requests More Data for Unique Osteoporosis Drug Candidate

By July 17, 2017

Dr. Pascale Richetta, head of bone and executive vice president, UCB, stated, "With all three pivotal romosozumab Phase 3 studies now included in the clinical evidence package, representing data from more than 11,000 patients, we are committed to bringing this important potential new treatment to those people living at risk of fragility fractures."

First-in-Class Drug for Primary Hyperoxaluria Gets Orphan Drug Designation

By July 13, 2017

The Orphan Drug designation was supported by data from preliminary studies showing that ALLN-177 significantly decreased urinary and plasma oxalate in several rodent and porcine models.

FDA Fast Tracks New Oral Antifungal to Treat Valley Fever

By July 13, 2017

VT-1598, an oral fungal CYP51 inhibitor, has shown high potency against pathogens such as molds, yeasts, and multi-drug resistant fungal pathogens (eg, Candida auris).

Cancer Peptide Vaccine Designated Orphan Drug for MDS

By July 10, 2017

Data from a Phase 1/2 study in patients with MDS who had disease progression on or after first-line azacitidine treatment showed initial signs of clinical activity with DSP-7888, and that it was well tolerated in study patients.

Advanced Breast Cancer Drug Granted Priority Review

By July 10, 2017

The NDA included data from two trials, MONARCH 1 and MONARCH 2, which are a part of the comprehensive MONARCH clinical trial program.

Entrectinib Gains Orphan Drug Status for NTRK Fusion(+) Tumors

By July 10, 2017

Currently, entrectinib is being evaluated in the global, multicenter, open-label, potentially registration-enabling Phase 2 STARTRK-2 trial (Studies of Tumor Alterations Responsive to Targeting Receptor Kinases)

Trevyent NDA Submitted for Pulmonary Arterial Hypertension

By July 06, 2017

Trevyent contain treprostinil, a vasodilatory prostacyclin analogue. It works primarily through vasodilation of pulmonary and systemic arterial vascular beds, and inhibition of platelet aggregation.

SOBI003 Gains Orphan Drug Status for Rare Metabolic Disorder

By July 05, 2017

According to the Company, SOBI003 is currently in the late pre-clinical phase and its first clinical trial is expected to commence in 2018.

Biomarin Submits BLA for New Phenylketonuria Therapy

By July 05, 2017

The investigational drug is intended to substitute the deficient PAH enzyme with a PEGylated phenylalanine lyase enzyme to break down Phe.

Astellas Seeks Approval for Overactive Bladder Combo Therapy

By June 30, 2017

"We look forward to FDA's review of our application for a potential new treatment option for the millions of people living with OAB," said Bernhardt Zeiher, MD, president of Development at Astellas.

Emicizumab Beneficial in Hemophilia A Patients With Inhibitors to FVIII

By June 29, 2017

The primary analysis of the study arm showed a statistically and clinically significant reduction in bleeding by 87% (risk rate [RR] 0.13; P<0.0001] with emicizumab prophylaxis.

Rituxan Biosimilar BLA Accepted for FDA Review

By June 29, 2017

The proposed indications are for the treatment of non-Hodgkin's lymphoma (NHL), chronic lymphocytic leukemia (CLL), rheumatoid arthritis (RA), granulomatosis with polyangiitis and microscopic polyangiitis.

New Antibiotic Designated QIDP for Two Indications

By June 29, 2017

The gel is hormone-free and can be used with other forms of contraception or used alone. The gel, supplied in a pre-filled applicator, can be self-applied.

NDA Resubmitted for Twirla Contraceptive Patch

By June 28, 2017

In the NDA resubmission, the Company included safety and efficacy data from the Phase 3 trial SECURE, the manufacturing information, and a summary response to the CRL.

Miltefosine Gains Orphan Drug Status for Serious CNS Infections

By June 28, 2017

Miltefosine is an alkyllysophospholipid analogue drug with in vitro activity against the promastigote and amastigote stages of Leishmania species. It is already approved to treat visceral, mucosal and cutaneous leishmaniasis in patients aged ≥12 years; it is the first oral treatment for this rare tropical parasitic disease.

FDA to Review Lower Dose Xarelto for Recurrent VTE Risk Reduction

By June 28, 2017

Both doses of Xarelto (10mg and 20mg) were superior to aspirin (100mg) in lowering the risk of recurrent VTE after ≥6 months of standard anticoagulation.

Novel CETP Inhibitor Shows Coronary Improvements in Phase 3 Study

By June 27, 2017

The full results of the REVEAL trial will be announced at the European Society of Cardiology meeting on August 29.

NDA Accepted for Anti-Inflammatory Inj Post-Cataract Surgery

By June 26, 2017

Dexycu injection was developed to help compliance—especially among elderly patients—and avoid dosing errors associated with conventional treatments that depend on self-administered eye drops multiple times a day.

Novel Synthetic Bio Drug for Hyperammonemia Granted Fast Track

By June 26, 2017

Synthetic Biotic medicines utilize synthetic biology to reprogram probiotic bacteria to perform critical functions that compensate for those missing or damaged due to a particular disease.

Upadacitinib Meets Endpoints for Phase 3 Study in RA

By June 23, 2017

The results from the 12-week period show that both arms of upadacitinib treatment were superior in achieving ACR 20 (64% in 15mg arm, 66% in 30mg arm) vs. placebo (36%).

Novel Liquid Soln for Lennox Gastaut Syndrome Gets Orphan Drug Status

By June 22, 2017

The Company is awaiting top-line results of the current trial before initiating the LGS study.

Investigational Opioid Use Disorder Injectable Tx Shows Promise

By June 21, 2017

Results showed that at Month 6, the RBP-6000 groups demonstrated abstinence rates of 41.3% (300/300mg dose) and 42.7% (300/100mg dose) vs. 5% for placebo (P<0.0001), a statistically significant difference.

First-in-Class Pancreatic Cancer Drug Gains Orphan Drug Status

By June 21, 2017

Clinical trials of pamrevlumab are currently ongoing for conditions such as idiopathic pulmonary fibrosis, pancreatic cancer, and Duchenne muscular dystrophy.

Shingrix Proves Noninferior to Current Zoster Vaccine in Study

By June 21, 2017

"[These results] indicate that Shingrix can be an option for adults over 50 years of age, who previously received the currently available vaccine and are seeking to benefit from revaccination with Shingrix, if recommended," said Dr. Thomas Breuer, SVP and CMO of GSK Vaccines.

FDA Fast Tracks Drug for Hallucinations Disorder in Lewy Body Dementia

By June 19, 2017

Nelotanserin is a novel once-daily oral 5HT2A inverse agonist. The 5HT2A receptor has been associated with neuropsychiatric effects, including visual hallucinations.

NDA Filed for Tavalisse for Immune Thrombocytopenia Treatment

By June 19, 2017

Tavalisse is an oral treatment designed to inhibit SYK kinase, a key signaling member in the immune process that leads to platelet destruction in ITP.

First-in-Class Drug Candidate Fast Tracked for Chronic Pain

By June 14, 2017

Currently, there is a global Phase 3 program comprised of 6 studies involving approximately 7,000 patients with OA, CLBP or cancer pain with inadequate treatment of pain with approved therapies.

Triple-Therapy, Single Tablet HIV Regimen Submitted to FDA for Review

By June 12, 2017

Three of the ongoing studies are evaluating the safety and efficacy of BIC/FTC/TAF vs. triple-therapy regimens containing dolutegravir 50mg among treatment-naive patients and among virologically-suppressed patients who are switching from an existing antiretroviral regimen with dolutegravir.

Galcanezumab Shown to Significantly Cut Monthly Migraine Days

By June 10, 2017

Patients in the galcanezumab group had statistically significant greater reduction in monthly migraine headache days with acute medication use compared to placebo.

FDA to Review Trulance for IBS-C Indication in Adults

By June 07, 2017

Results from both studies showed Trulance was statistically superior to placebo in achieving the primary endpoint (Study 1: 30.2%, P<0.001 in 3mg and 29.5% in 6mg vs. 17.8% in placebo; Study 2: 21.5% in 3mg and 24.0% in 6mg vs. 14.2% in placebo).

JZP-110 Effective for Excessive Sleepiness in OSA Patients

By June 06, 2017

Significant overall improvements were also noted on the key secondary endpoint of improved Patient Global Impression of Change (PGIc) scale, with 90% of patients in the 150mg and 300mg arms reporting overall improvement at Week 12.

Tafamidis Gets Fast Track Status for Rare Disease Linked to Progressive HF

By June 06, 2017

Vyndaqel is approved in Europe and a number of other countries to treat transthyretin familial amyloid polyneuropathy (TTR-FAP) but not in the U.S.

FDA to Review New Evolocumab Cardiovascular Outcomes Data

By June 05, 2017

Results showed that the Repatha plus statin group had a statistically significant 20% (P<0.001) reduction in hard major adverse cardiovascular events.

Inhaled L-dopa Meets Endpoints in Parkinson's Disease Study

By June 05, 2017

The study's primary endpoint was meaningful improvement in motor function as defined by the Unified Parkinson's Disease Rating Scale—Part III.

FDA to Review Opdivo for Hepatocellular Carcinoma Indication

By June 02, 2017

The sBLA submission was based on results from the Phase 1/2 CheckMate -040 study.

Vyxeos Granted Priority Review by FDA

By May 31, 2017

Vyxeos, or CPX-351, combines cytarabine and daunorubicin in a nano-scale liposome at a 5:1 molar ratio using the CombiPlex platform.

CGRP Targeted Drug Looks Promising for Migraine Prevention

By May 31, 2017

The HALO Clinical Research Program consists of two 16-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group studies that compared fremanezumab to placebo in adults with episodic and chronic migraine.

Acute Hepatic Porphyria Therapy Gets Breakthrough Designation

By May 31, 2017

Currently, the only approved treatment for AHP attacks is hemin for injection (Panhematin or Normosang).

Priority Review Granted for Axicabtagene Ciloleucal in NHL

By May 26, 2017

In the ZUMA-1 trial 42% of patients demonstrated ongoing response at the 8.7-month follow-up, of which 39% achieved complete response.

Lurasidone Benefits Younger Patients with Bipolar Depression in Phase 3 Study

By May 24, 2017

Results showed a statistically significant change from baseline to Week 6 in favor of Latuda vs. placebo in the Clinical Global Impression-Bipolar Version, Severity of Illness (CBI-BP-S) score and the Children's Depression Rating Scale, Revised (CDRS-R) score.

New CRAC Channel Inhibitor for Acute Pancreatitis Fast-Tracked

By May 24, 2017

In Phase 1 safety studies, CM4620 showed positive activity in vivo in several animal models. CalciMedica plans to begin the first of its patient studies early in 2018.

Tildrakizumab to Be Reviewed for Moderate-to-Severe Plaque Psoriasis

By May 24, 2017

Ongoing data from two Phase 3 trials, ReSurface 1 and ReSurface 2, which enrolled over 1,800 patients, is included in the BLA.

Novel HIV Vaccine Shows High Immune Response Rate in Study

By May 24, 2017

Overall, 71 of 76 participants (93%) showed detectable CD4+ or CD8+ responses to at least one of the vaccine's four antigens.

P2X3 Receptor Antagonist Investigated for Chronic Cough

By May 24, 2017

Results, measured by a recording device, found that those in the 50mg MK-7264 group had a statistically significant reduction in cough frequency of 37% when compared with placebo.

Roclatan Meets Primary Endpoint in Phase 3 Glaucoma Study

By May 24, 2017

The evaluation of Roclatan has been ongoing in two distinct trials.

Sinuva Implant for Chronic Sinusitis to Be FDA Reviewed

By May 23, 2017

The Sinuva implant releases mometasone furoate to the ethmoid sinus lining and is intended to treat chronic sinusitis patients who have recurrent sinus obstruction.

Novel Oral Therapy Looks Promising for Relapsing MS

By May 22, 2017

The Radiance trial included 1,313 relapsing multiple sclerosis (RMS) patients who were administered either 0.5mg or 1mg of oral ozanimod, or weekly intramuscular interferon beta-1a (Avonex) over a 2 year treatment period.

Benralizumab Use Cuts Need for Oral Steroids in Severe Asthma

By May 22, 2017

The primary endpoint was met with 75% of the benralizumab group reducing their OCS doses compared to 25% in the placebo group. Additionally, 52% of the benralizumab group who had optimized baseline OCS doses of ≤12.5mg/d were able to completely stop OCS use.

Aptiom sNDA for Pediatric Indication Accepted for Review

By May 22, 2017

The sNDA submission includes data from five controlled clinical trials in adults with POS and three clinical studies demonstrating safety and tolerability in pediatric patients.

Biologic for Migraine Prevention Submitted to FDA

By May 18, 2017

Amgen announced the submission of a Biologics License Application (BLA) to the Food and Drug Administration (FDA) for erenumab for the prevention of migraines.

Neuroactive Steroid Gets Fast Tracked for Major Depressive Disorder

By May 18, 2017

The Food and Drug Administration (FDA) has granted Fast Track designation to SAGE-217 (Sage Therapeutics) for the potential treatment of major depressive disorder (MDD).

Copanlisib Granted Priority Review for Follicular Lymphoma

By May 17, 2017

Orphan designation has also been granted to copanlisib for the treatment of splenic, nodal, and extranoldal subtypes of marginal zone lymphoma.

AML Drug Candidate Granted Breakthrough Therapy Designation

By May 17, 2017

Data from an ongoing multicenter, open-label Phase 1/2 trial was presented at the American Society of Hematology in 2016 that evaluated GMI-1271 as adjunct to chemotherapy to patients with relapsed/refractory AML in addition to patients aged ≥60 years with newly diagnosed disease.

Fast Track Status Granted to Tideglusib for Myotonic Dystrophy

By May 17, 2017

In preclinical studies, AMO-02 has shown efficacy in transgenic models and reversal of muscle cell differentiation deficits in ex vivo tissue samples.

Novel Hemophilia A Gene Therapy Gets Fast Track Status

By May 16, 2017

The Food and Drug Administration (FDA) has granted Fast Track designation to SB-525 (Sangamo Therapeutics) for the treatment of hemophilia A.

Inotersen Shows Promise in Phase 3 Familial Amyloid Polyneuropathy Study

By May 15, 2017

Ionis announced positive results from the Phase 3 study of inotersen (IONIS-TTRRx) for the treatment of familial amyloid polyneuropathy (FAP), now referred to as hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN).

FDA Accepts NDA for Glaucoma Drug Rhopressa

By May 15, 2017

The FDA has completed its initial 60-day review of the New Drug Application (NDA) for Rhopressa (netarsudil; Aerie) 0.02% and determined that it's sufficiently complete to permit a substantive review.

Trigriluzole Fast Tracked for Rare Neurodegenerative Disorder

By May 15, 2017

The FDA has granted Fast Track designation to trigriluzole (BHV-4157; Biohaven) for the potential treatment of Spinocerebellar Ataxia (SCA), a rare and debilitating neurodegenerative disorder.

Tenapanor Achieves Primary Endpoint in IBS-C Trial

By May 12, 2017

Results showed that the endpoint was met with 27% in the tenapanor arm compared to 18.7% in the placebo arm (P=0.02) having at least 30% reduction in abdominal pain and an increase of 1 or more complete spontaneous bowel movements.

Novel Treatment for IgG4-Related Disease Gains Orphan Drug Status

By May 12, 2017

There is currently no approved treatment for lgG4-RD, which is a newly recognized disorder and is estimated to affect 40,000 individuals in the U.S.

Once-Monthly Biologic Looks Promising for Migraine Prophylaxis

By May 12, 2017

Lilly announced encouraging results from three Phase 3 studies of galcanezumab, EVOLVE-1, EVOLVE-2 and REGAIN, evaluating its efficacy and safety for the prevention of episodic and chronic migraine.

First-in-Class Oral Enzyme Designated Breakthrough Therapy for CDI Prevention

By May 11, 2017

The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for ribaxamase (SYN-004; Synthetic Biologics) for the prevention of Clostridium difficile infection (CDI).

Gimoti Shows Benefit in Moderate to Severe Diabetic Gastroparesis

By May 11, 2017

Results showed that the primary efficacy endpoint of change from baseline to week 4 was not statistically met (P=0.881), although a significant treatment benefit was seen in patients with moderate to severe symptoms (P<0.05).

FDA Grants Vonapanitase Breakthrough Therapy Status for Hemodialysis Use

By May 10, 2017

A second Phase 3 trial for vonapanitase in hemodialysis patients, PATENCY-2, is currently ongoing and is expected to complete enrollment in the first quarter of 2018.

FDA Issues Complete Response Letter for Dyspareunia Drug

By May 09, 2017

The FDA has issued a Complete Response Letter (CRL) to the New Drug Application (NDA) of TX-004HR (TherapeuticsMD) for the treatment of moderate-to-severe vaginal pain during sexual intercourse (dyspareunia), a symptom of vulvar and vaginal atrophy (VVA) due to menopause.

FDA Fast Tracks Gene Therapy for Chronic Angina Patients

By May 08, 2017

XC001 is an investigational cardiovascular angiogenic gene therapy that promotes angiogenesis to provide arterial blood flow to myocardial regions with inadequate blood supply.

FDA Fast Tracks Metastatic Breast Cancer Combo Treatment

By May 08, 2017

The only adverse event that was grade 3 or higher was fatigue with 16.3% incidence in the treatment group and 13.2% in the control group.

Xeljanz Under FDA Review for Psoriatic Arthritis

By May 05, 2017

An anticipated Prescription Drug User Fee Act (PDUFA) action date of December 2017 has been set by the FDA for both Xeljanz sNDAs.

Brexpiprazole Evaluated for Agitation in Alzheimer's Dementia

May 03, 2017

Otsuka and Lundbeck announced top-line results from two Phase 3 trials of brexpiprazole for the treatment of agitation in patients with dementia of the Alzheimer's type.

Xarelto sNDA Looks to Add New Dosing Option to Reduce Recurrent VTE Risk

By May 02, 2017

The new sNDA submission is supported by the 'Einstein Choice' study, which included 3,365 patients in a Phase 3 randomized, double-blind study that compared Xarelto once daily to aspirin.

Intranasal Midazolam Evaluated as Rescue Therapy for Seizure Clusters

By May 02, 2017

Proximagen announced promising data from the pivotal Phase 3 trial of USL261 (intranasal midazolam) for the rescue treatment of seizure clusters. The primary efficacy endpoint was treatment success, defined as achieving 1) termination of seizure(s) and 2) no recurrence of seizure(s)