Treatment for Rare Pediatric Cancer Granted Orphan Drug Designation

By May 10, 2018

CLR 131 is an investigational radioiodinated Phospholipid Drug Conjugate (PDC) drug candidate that delivers cytotoxic radiation directly and selectively to malignant cells.

Vaccine for Chikungunya Prevention Gets Fast Track Designation

By May 04, 2018

There are currently no approved treatments or vaccines available for chikungunya, a virus transmitted through mosquito bites.

Facioscapulohumeral Muscular Dystrophy Treatment Gets Fast Track Status

By May 02, 2018

ACE-083 is currently being studied in a Phase 2 trial for FSHD and another for Charcot-Marie-Tooth (CMT) disease.

First-in-Class Antifungal Fast-Tracked for Vulvovaginal Candidiasis

By May 01, 2018

SCY-078 is an investigational antifungal agent that is a semi-synthetic derivative of the natural product enfumafungin.

Opsumit sNDA Submitted for Chronic Thromboembolic Pulmonary Hypertension

By April 30, 2018

The sNDA is accompanied by results from the MERIT-1 trial, a Phase 2 randomized, placebo-controlled, double-blind study which assessed the efficacy, safety and tolerability of macitentan 10mg in 80 patients with inoperable CTEPH.

Results From Phase 3 Trial of Migraine Tx Ubrogepant Released

By April 27, 2018

Results showed that 14.3%, 20.7% (P=.0285), and 21.8% (P=.0129) achieved pain freedom in the placebo, ubrogepant 25mg dose, and 50mg dose groups, respectively.

FDA to Review Fluzone Quadrivalent 0.5mL Dose for Younger Patients

By April 27, 2018

The sBLA included clinical data from a Phase 4 study that enrolled nearly 2000 children and assessed the safety and immunogenicity of the 0.5mL dose.

FDA Fast Tracks Gene Therapy for X-Linked Retinitis Pigmentosa

By April 27, 2018

XLRP represents the most severe form of retinitis pigmentosa (RP), an inherited retinal disease characterized by progressive retinal degeneration and vision loss that ends in complete blindness.

FDA Grants Fast Track Status to IBS-D Treatment

By April 26, 2018

ORP-101 is a peripherally active partial agonist of the μ opioid receptor and antagonist of the κ opioid receptor.

Risankizumab BLA Submitted for Moderate to Severe Plaque Psoriasis

By April 25, 2018

The BLA is supported by data from the global risankizumab Phase 3 psoriasis program evaluating more than 2,000 patients.

NDA Submitted for Postpartum Depression Treatment Brexanolone

By April 23, 2018

Results in the 202B and 202C trials showed that brexanolone achieved the primary endpoint of mean reduction from baseline in the Hamilton Rating Scale for Depression.

MenB Vaccine Gets Breakthrough Tx Designation for Kids Ages 1 to 9 Years

By April 23, 2018

The Breakthrough designation was based on data from Phase 2 studies which assessed the safety and efficacy of the vaccine in this patient population.

Gene Therapy Looks Promising for Transfusion-Dependent β-Thalassemia

By April 19, 2018

The safety and efficacy of LentiGlobin were evaluated in 2 independent 2-year clinical studies: HGB-204 (Northstar, N=18), an open-label, single-dose, nonrandomized, multicenter Phase 1/2 study in patients with TDT, and HGB-205, an ongoing open-label, single-dose, nonrandomized, single-center Phase 1/2 study in patients with TDT and severe sickle cell disease.

Oraxol Gets Orphan Drug Designation for Angiosarcoma

By April 19, 2018

Oraxol is a novel oral therapy that combines paclitaxel, a tubulin-stabilizing chemotherapeutic agent, with a non-absorbable gastrointestinal tract P-glycoprotein pump inhibitor.

Hemlibra Gets Breakthrough Tx Status for Hemophilia A Without Inhibitors

By April 18, 2018

The trial enrolled 152 patients with hemophilia A who were previously treated, either on-demand or as prophylaxis, with factor VIII therapy.

Esketamine Evaluated in MDD Patients at Imminent Risk for Suicide

By April 16, 2018

If approved, esketamine has the potential to be the first treatment for patients with major depressive disorder at imminent risk for suicide.

FDA to Review Opioid System Modulator for Major Depressive Disorder

By April 16, 2018

ALKS 5461 consists of a fixed-dose combination of buprenorphine, a partial mu-opioid receptor agonist and kappa-opioid receptor antagonist, and samidorphan, a mu-opioid receptor antagonist.

Long-Term Obeticholic Acid Linked to Fibrosis Regression in PBC Patients

By April 13, 2018

Results showed that 46% (n=6) of substudy patients improved their histological fibrosis stage, while 38% maintained (n=5), and 15% experienced 1 stage progression (n=2).

Treatment for Idiopathic Pulmonary Fibrosis Gets Orphan Drug Designation

By April 10, 2018

RP5063 is a new chemical entity with a novel mechanism of multimodal modulation of serotonin and dopamine signaling pathways.

Phase 3 Results Released for Investigational Alzheimer's Disease Treatment

By April 10, 2018

Results showed that after 18-months, there was no significant statistical difference in cognitive or functional outcomes between those patients taking azeliragon and the placebo group.

Top-Line Results Announced for Investigational RA Treatment Upadacitinib

By April 09, 2018

At week 12, results showed 71% in the upadacitinib group had achieved ACR20, compared to 63% and 36% in the adalimumab and placebo groups, respectively.

Elamipretide Granted Orphan Drug Status for Barth Syndrome

By April 09, 2018

The TAZPOWER study is a randomized, double-blind, placebo-controlled Phase 2/3 crossover study evaluating the safety and efficacy of elamipretide (daily subcutaneous injections) in 12 male patients (aged ≥12 years) with genetically-confirmed Barth syndrome.

Duvelisib NDA Gets Priority Review for Relapsed/Refractory CLL/SLL, Follicular Lymphoma

By April 09, 2018

Duvelisib is a first-in-class oral dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma.

Tenalisib Granted Orphan Drug Designation for Cutaneous T-Cell Lymphoma

By April 09, 2018

Preclinical studies have demonstrated that RP6530 reprograms macrophages from an immunosuppressive M2-like phenotype (pro-tumor) to an inflammatory M1-like state (anti-tumor), which can potentially enhance the activity of checkpoint inhibitors or overcome resistance to these agents.

Omadacycline Granted Priority Review for CABP, Skin/Skin Structure Infections

By April 05, 2018

Omadacycline is an investigational, once-daily, broad-spectrum aminomethylcycline antibiotic that is related to tetracyclines.

Dacomitinib Granted Priority Review for NSCLC With EGFR-Activating Mutations

By April 04, 2018

Dacomitinib is a pan-human epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor.

Positive Top-Line Results for Ramucirumab in HCC Announced

By April 04, 2018

REACH-2 was a randomized, double-blind, placebo-controlled trial that enrolled 292 patients with hepatocellular carcinoma (HCC) who were intolerant to, or had disease progression while on or following treatment with sorafenib and had a high alpha-fetoprotein (AFP-High) defined as AFP ≥400ng/mL.

Novel Cystic Fibrosis Treatment Granted Fast Track Status

By April 04, 2018

Proteostasis Therapeutics is developing the combination treatment which includes a novel transmembrane conductance regulator (CFTR) amplifier (PTI-428), a third generation corrector (PTI-801) and a potentiator (PTI-808).

First-in-Class Treatment for Hairy Cell Leukemia Under FDA Priority Review

By April 03, 2018

Moxetumomab pasudotox is an investigational anti-CD22 recombinant immunotoxin.

Firdapse NDA Submitted for Lambert-Eaton Myasthenic Syndrome

By April 02, 2018

Results from the Phase 3 trial showed that the Quantitative Myasthenia Gravis (QMG) score at baseline to day 14 changed from 6.4 to 6.7 in the amifampridine group vs. 5.6 to 7.9 for the placebo group.

Novel Nasal Spray for Migraine Attacks to Be FDA Reviewed

By April 02, 2018

DFN-02 was evaluated in a multicenter, double-blind, randomized, placebo-controlled study (N=107) which showed it was effective in treating pain and associated symptoms during a migraine attack and in reducing attack-related functional disability.

NDA Submitted for Sublingual Film to Treat OFF Episodes in Parkinson's Disease

By March 30, 2018

The NDA submission included data from Phase 3, 12-week, randomized, double-blind, placebo-controlled, parallel-group study (CTH-300) that enrolled patients with levodopa-responsive Parkinson's disease complicated by OFF episodes.

Oral Tx for Transthyretin Cardiomyopathy Meets Primary Endpoint in Study

By March 30, 2018

Although they acknowledge the preliminary data is subject to further analysis, they say it demonstrates a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo at 30 months.

Livantra Granted Orphan Drug Status for Acute-on-Chronic Liver Failure

By March 30, 2018

Livantra may help rate of survival without transplantation or help improve survival until patients can receive a new liver.

Unique Vit B3 Formulation May Improve Blood Pressure, CV Health

By March 29, 2018

NR supplementation showed reductions in mean systolic blood pressure (SBP; mean change -3.9mmHg) and diastolic blood pressure (DBP; mean change -2.0mmHg) for all patients; these findings were not statistically significant after adjusting for multiple comparisons.

NDA for Novel Amikacin Inhalation Submitted for Rare Lung Infection

By March 29, 2018

ALIS has previously been designated orphan drug status, breakthrough therapy status and a Qualified Infectious Disease Product.

First-in-Class Inhibitor Designated Orphan Drug for Spinal Muscular Atrophy

By March 29, 2018

By inhibiting the activation of myostatin, SRK-015 may promote a clinically meaningful increase in muscle mass and strength.

Bremelanotide NDA Submitted to FDA for Hypoactive Sexual Desire Disorder

By March 26, 2018

Bremelanotide is used as needed prior to anticipated sexual activity.

Sotagliflozin NDA Submitted to FDA for Type 1 Diabetes

By March 26, 2018

Sotagliflozin is an investigational dual sodium-glucose co-transporter types 1 and 2 (SGLT1, SGLT2) inhibitor.

Urothelial Cancer Treatment Granted FDA Breakthrough Therapy Designation

By March 26, 2018

Enfortumab vedotin is an investigational ADC composed of an anti-Nectin-4 monoclonal antibody attached to monomethyl auristatin E, a microtubule-disrupting agent.

Siponimod Reduces Risk of Disability Progression in Secondary Progressive MS

By March 23, 2018

Siponimod is an investigational, selective modulator of specific subtypes of the sphingosine-1-phosphate (S1P) receptor.

FDA Accepts Durasert NDA for 3-Year Treatment of Posterior Segment Uveitis

By March 22, 2018

The FDA have set a Prescription Drug User Fee Act (PDUFA) date of November 5, 2018 for Durasert.

FDA Fast Tracks Pedmark for Prevention of Cisplatin-Induced Ototoxicity

By March 21, 2018

The Clinical Oncology Group Protocol ACCL0431 trial enrolled 125 pediatric patients newly diagnosed with a germ cell tumor, hepatoblastoma, medulloblastoma, neuroblastoma, osteosarcoma, or other malignancy.

Nintedanib Fast Tracked for ILD-Associated Scleroderma

By March 19, 2018

The designation was supported by the Investigational New Drug application (IND) for the treatment of SSc-ILD and the anticipated safety and efficacy data from the Phase 3 double-blind, randomized, placebo-controlled SENSCIS trial.

Treatment for Fragile X Syndrome Gets Fast Track Status

By March 15, 2018

A Phase 2 trial of OV101 in males with Fragile X syndrome aged 13 to 22 is planned for later in 2018. OV101 has previously been granted Orphan Drug status for Fragile X and Fast Track status for Angelman syndrome.

Cabometyx sNDA for Hepatocellular Carcinoma Indication Submitted to FDA

By March 15, 2018

CELESTIAL is a randomized, double-blind, placebo-controlled study of cabozantinib in patients with advanced HCC conducted at more than 100 sites globally in 19 countries.

FDA to Review Xeomin for the Management of Excessive Drooling

By March 14, 2018

The sBLA is based on a Phase 3, randomized, double-blind, placebo-controlled, multicenter 184 subject trial.

Positive Results for Elagolix in Second Phase 3 Uterine Fibroid Trial

By March 14, 2018

Results - measured by the alkaline hematin method - found that at 6 months those in the combination group had 76.2% (P<0.001) clinical response compared to the elagloix along group, in which only 10.1% had a clinical response.

Sollpura Falls Short in Exocrine Pancreatic Insufficiency Trial

By March 13, 2018

the Phase 3 'SOLUTION' study of Sollpura did not meet the primary endpoint of non-inferiority to an active-comparator in the coefficient of fat absorption (CFA).

FDA Accepts NDA for Bio-Identical Combination Hormone Therapy

By March 09, 2018

TX-001HR is an investigational treatment for moderate-to-severe vasomotor symptoms due to menopause.

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.