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.

Phase 3 Results Announced for Lusutrombopag for Thrombocytopenia

By September 25, 2017

The trial, L-PLUS2, included 215 patients who were randomized 1:1 to either 3mg of lusutrombopag or placebo for up to 7 days.

FDA Rejects Vraylar sNDA for Negative Symptoms of Schizophrenia

By September 25, 2017

In the letter the FDA stated that their preliminary appraisal found that the sNDA was not sufficiently complete to permit a substantive review.

NDA for First Darunavir-Based Single-Tab HIV Regimen Submitted

By September 25, 2017

The treatment will become the first darunavir-based, single-tablet HIV treatment, if approved.

NDA Submitted for Thrombocytopenia Treatment

By September 22, 2017

The results from Phase 3 trials demonstrated efficacy and safety in patients with severe thrombocytopenia and with CLD.

UTI Treatment Gains Qualified Infectious Disease Product Designation

By September 21, 2017

"We expect to begin enrolling patients as early as December in a proof-of-concept Phase 2 study of omadacycline in uUTI, and an indication in this type of infection would broaden the opportunities for oral omadacycline in community-acquired infections," said Evan Loh, MD, COO and CMO of Paratek.

Treatment for Subtype of T-Cell Lymphoma Granted Breakthrough Designation

By September 20, 2017

The study enrolled a total of 372 patients with MF and SS who were randomized to either receive mogamulizumab or vorinostat.

Orphan Drug Designation Granted to ALS Treatment

By September 19, 2017

A Phase 2 randomized, double-blind, placebo-controlled trial of AMX0035 titled CENTAUR (nct03127514) has begun with 132 ALS participants enrolled.

Phase 3 Results of Novel CABP Treatment Announced

By September 18, 2017

A second 'LEAP 2' trial is currently in the midst of enrolling patients and expected to have topline results in the spring of 2018.

Investigational Migraine Tx Shows Promise in Phase 3 Trial

By September 14, 2017

Results demonstrated that at 12 weeks after the first dose of fremanezumab, the CM group had reductions of monthly headaches; -4.6 days for the monthly dose, -4.3 days for the quarterly dose and -2.5 for the placebo group (P<0.0001).

FDA Committee Gives Thumbs Up to New Shingles Vaccine Shingrix

By September 13, 2017

While not bound by the Advisory Committee's recommendation, the FDA takes its guidance into consideration.

Cabometyx sNDA Submitted for Expanded Renal Cell Carcinoma Indication

By September 13, 2017

"If [the sNDA is] approved, Cabometyx will offer an important new alternative for the treatment of patients with previously untreated advanced RCC," said Michael M. Morrissey, PhD, President and CEO of Exelixis.

Romosozumab Followed by Alendronate Superior in Reducing Fracture Risk

By September 12, 2017

ARCH (Active-contRolled FraCture Study in Postmenopausal Women with Osteoporosis at High Risk of Fracture) was a multicenter, international, randomized, double-blind, alendronate-controlled study (n=4,093) in postmenopausal women with osteoporosis at high risk for fracture based on previous fracture history.

Dupilumab Beneficial in Patients with Uncontrolled, Persistent Asthma

By September 12, 2017

Results found that at 52 weeks severe asthma attacks were reduced by 46% in the overall population for the 300mg dose group and by 60% and 67% among those patients with ≥150 eosinophilic cells/microliter or greater and ≥300 eosinophilic cells/microliter, respectively (P<0.001).

Cemiplimab Gains Breakthrough Tx Designation for Advanced Skin Cancer

By September 08, 2017

A Phase 2, single-arm, open-label clinical trial, EMPOWER-CSCC 1, is now enrolling patients with metastatic CSCC and locally advanced and unresectable CSCC.

Pediatric Ulcerative Colitis Tx Granted Orphan Drug Designation

By September 08, 2017

Early Phase 1 data has indicated that the treatment drug can be more efficacious than mesalamine/5-ASA treatments alone.

Oral Tx for Acute Suicidal Ideation and Behavior Gets Fast Track Status

By September 06, 2017

Results from two previously Phase 2 trials of NeuroRx's sequential treatment have shown a 50% reduction in symptoms of depression and a 75% reduction in suicidal ideation in bipolar patients.

Elagolix NDA Submitted for Treating Endometriosis-Associated Pain

By September 06, 2017

Elagolix, an oral gonadotropin-releasing hormone (GnRH) antagonist, was evaluated in two Phase 3 clinical studies in about 1,700 women with moderate-to-severe endometriosis-associated pain.

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.

Two BLAs for Herceptin Biosimilars Submitted to FDA

By July 31, 2017

Two separate Biologics License Applications (BLAs) for biosimilar candidates to Herceptin (trastuzumab) have been submitted to the Food and Drug Administration (FDA).

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.