The FDA approved emapalumab (Gamifant from Novimmune) on 11/20/18 for the treatment of refractory, recurrent, or progressive primary hemophagocytic lymphohistiocytosis (HLH) or intolerance to conventional HLH therapy.
The FDA approved glasdegib (Daurismo from Pfizer) on 11/21/18 for the treatment of newly diagnosed acute myeloid leukemia (AML) in patients aged 75 years or older or patients that are not candidates for intensive chemotherapy.
The FDA delayed a decision on Sage Therapeutics’ brexanolone by three months to work on a REMS program for the drug. The FDA did not ask for any additional safety or efficacy data. The new PDUFA Date is May 19, 2019, this drug has Orphan Drug and Breakthrough Priority Designations.
In a Phase III trial, 67% of patients that received Aimmune Therapeutics’ AR101 for peanut allergy were able to tolerate a 600mg dose of peanut protein compared to 4% with placebo and 50% of patients tolerated 1000 mg of peanut protein compared to 2.4% with placebo. 90% of patients were < 18. There was no improvement in peanut protein tolerance in patients > 17. Severe reactions were seen in 4.3% of the AR101 group vs. 0.8% with placebo and 11.6% of AR101 patients withdrew from the trial due to ADR compared to 2.4% with placebo with 14% of AR101 patients requiring epinephrine during the study.
Novo announced that in a Phase III trial, semaglutidereduced a combined endpoint of cardiovascular death, non-fatal myocardial infarction or non-fatal stroke compared to placebo when added to standard care in patients with type 2 diabetics. Novo announced that in another Phase III trial, daily oral semaglutide HbA1c was lowered by 1.7% with 14mg, 1.5% with 7mg and 1.7% with 3 mg compared to a 1.4% reduction with daily liraglutide 0.9 mg in Japanese type 2 diabetic patients. Only the 14 mg dose was statistically superior to liraglutide.
Blueprint Medicines announced interim results from an open label Phase I trial where treatment with avapritinibresulted in an 84% objective response rate (ORR) in patients with gastrointestinal stromal tumors and a PDGFRA mutation compared to a 25% ORR in patients without the mutation.
Northwest Biotherapeutics’ DCVax-L is a cancer vaccine in development intended to prevent Glioblastoma multiforme. In a phase I/II trial 33% of patients had reached or exceeded the 4-year survival. Interim results from a 331 patient, Phase III trial suggested a survival benefit in patients treated with DCVax-L. Experts question if a conclusion can be drawn from the trial when completed, because patients were allowed to crossover if there was tumor progression. Almost 90% of patients received the vaccine. Enrollment was completed in 2015, but the trial will remain ongoing until the specified number of events have occurred. At two years, overall survival was 23.1 months with 46.4% of patients still alive after 24 months.
The FDA approved Salix/Cosmo Pharmaceuticals’ rifamycin (Aemcolo), on 11/17/18 for the treatment of adults with travelers’ diarrhea. The drug had FDA Qualified Infectious Disease Product and Fast Track priority designations.
Jazz submitted an MAA for solriamfetol in November 2018.
In a Phase II trial, NGM’s FGF19 Biosynthetic ngm282 did not change alkaline phosphatase compared to placebo in patients with Primary Sclerosing Cholangitis. Secondary endpoints suggested a decrease in fibrosis biomarkers. The drug has Orphan Drug and Fast Track priority designations but no PDUFA Date.
Mesoblast’s rexlemestrocel-L (MPC-06-ID) is being developed for the treatment of chronic low back pain (CLBP) due to disc degeneration. The FDA has granted rexlemestrocel-L an Orphan Drug priority designation.
Lilly filed an NDA for lasmiditan in November 2018. See yesterday’s Migraine update for more details about this ditan. https://www.prescriberight.com/pipeline-news/new-drugs-in-development-for-the-treatment-of-acute-migraines
BioCardia announced preliminary 12-month results from the first 10 patients in the Phase III CardiAMP-HF Trial. These patients, who had experienced heart failure following a myocardial infarction, demonstrated a non-significant improvement in their Six Minute Walk Distance.
Merck’s Ebola vaccine was developed by the Canadian Public Health Agency. The vaccine is licensed to NewLink Genetics, which partnered with Merck for promotion and sales. The vaccine is being used in a 2018 Ebola outbreak in the Democratic Republic of the Congo. Merck initiated a rolling NDA in November 2018 and plans to complete the filing in 2019.
In a Phase II trial, 32.6% of patients treated with Aurinia Pharmaceuticals’ voclosporin 23.7 mg and 27.3% of patients treated with voclosporin 39.5 mg achieved complete renal remission (CRR) compared to 19.3% with placebo in lupus nephritis patients treated with mycophenolate mofetil and rapidly tapered low-dose oral corticosteroids. At 48 weeks, CRR was significantly greater with low-dose voclosporin compared to placebo.
An analysis by WHO cautioned of an increased risk for severe dengue in seronegative vaccine recipients compared to seronegative non-vaccinated patients with Sanofi's dengue vaccine, but long-term protection in seropositive individuals is achieved.
An 18-patient, Phase II trial suggested that prostate-specific membrane antigen (PSMA) positron emission tomography (PET) may predict treatment response of Lu-PSMA-617.
In a 292 patient, phase IIb trial, Johnson & Johnson (Janssen)’s pimodivir decreased the influenza viral load with or without osteltamivir and there was a non-significant trend in symptom improvement with the two drugs combined compared to placebo. Primodivir monotherapy resolved symptoms similar to osteltamivir or the combination of the two. FDA granted Fast Track priority to primodivir.
Chugai Pharma/Genentech’s emicizumab reduced the number of bleeds by 87% compared to on-demand bypassing agents in the 109 hemophilia A patient, phase III HAVEN-1 trial. 63% of emicizumab patients had no bleeds over 31 weeks compared to 6% that received bypassing agents. The company also revealed that interim results from the phase III Haven-2 pediatric trial suggested similar results in children. In the HAVEN-1 trial, prophylaxis with emicizumab was associated with improvements in hemophilia patient health-related outcomes (Haem-A-QoL, Haemo-QoL SF, IUS, EQ-VAS). The drug’s PDUFA Date is February 23, 2019 and it has a Breakthrough Therapy priority designation.
In a 28-day, 38 patient, Phase II trial, treatment with Novartis/Conatus’ emricasan decreased ALT, cCK18, flCK18 and caspase compared to placebo in patients with non-alcoholic fatty liver disease. The FDA granted emricasan Orphan Drug and Fast Track priority designations.
CytoDyn announced viral suppression responder rates for patients treated with various doses of weekly subcutaneous leronlimab as monotherapy for at least 12 weeks as 44% with 350 mg, 71% with 525 mg and 92% with 700 mg in patients with HIV.
The brand name for RedHill Biopharma’s ABC294640 is Yeliva and the generic name is opaganib. The drug targets refractory or relapsed multiple myeloma and has an Orphan Drug priority designation.
AZ announced that in the phase III MYSTIC trial, tremelimumab plus durvalumab did not improve progression free survival compared to a platinum-based regimen in treating NSCLC. Tremelimumab has an Orphan Drug priority designation.
CHMP has recommended approval of fexinidazole.
United Therapeutics licensed marketing rights to ralinepag, from Arena Pharmaceuticals in November 2018.
Three new drugs are in development for the treatment of acute migraine. While many patients receive relief with triptans, not all patients can tolerate a triptan or are helped by a triptan. Clinical trials suggest that triptans relieve migraines in two hours in 16–51% of patients. The number of patients helped is likely higher since patients take a triptan dose after the pain is already moderate to severe, rather than early in the onset of the migraine attack in the trials. The triptans also have a contraindication in patients with cardiovascular disease, peripheral vascular disease, cerebrovascular disease, and uncontrolled hypertension because of the potential for ischemia.
For these reasons new drugs are being developed. The two classes of investigational migraine drugs include gepants (ubrogepant and rimegepant) and lasmiditan (a ditan). Neither class causesvasoconstriction, which removes safety concerns over ischemia. Two Phase III trials have been completed for each drug, but the results have not been published for any of the studies.
Lasmiditan(Lilly) is a highly selective 5-HT1 receptor agonist that inhibits central and peripheral neuronal activity without vasoconstriction. Ina 1,856 patienttrial, at 2 hours 59% of lasmiditan patients were pain free and 41% were free of their most bothersome symptoms compared to 43% for pain resolution and 30% for symptom resolution with placebo. In a2,155-patienttrial, 29% of patients treated with lasmiditan 50 mg, 31% with 100 mg and 39% with 200 mg were pain free at two hours compared to 21% with placebo. The most bothersome symptoms at two hours were eliminated in 41% oflasmiditan 50 mgpatients, 44% with 100 mg and 49% with 200 mg compared to 33% with placebo.
The other drug class being developed to treat acute migraine attacks are the gepants. Early gepants demonstrated liver enzyme elevation, so hepatic adverse events are continuing to be monitored as the gepants are developed. Each drug has been evaluated in two phase III trials and does not appear to cause hepatotoxicity.
Allergan purchased ubrogepant from Merck has continued its development for the treatment of migraine headaches. In a 1,327 patient, trial, at two hours 19% of migraine patients treated with ubrogepant 50 mg and 21% with 100 mg were pain free at two hours and 39% of patients were rid of their most bothersome symptoms with ubrogepant 50 mg and 38% with 100 mg compared to 12% and 29% with placebo. In a 1,355 patient, Phase III trial, the percentage of patients that were pain free at two hours were 21% with ubrogepant 25 mg, 22% with 50 mg and 14.3% with placebo. The most bothersome symptoms at two hours were eliminated in 39% of patients treated with ubrogepant 50 mg compared to 27% with placebo. The lower 25 mg dose of ubrogepant did not reach a statistical difference compared to placebo for freedom of the most bothersome symptoms.
Biohaven Pharmaceuticals bought rimegepantfrom BMS and continued its development. In a 1,084 patient, Phase III trial, 19% of patients treated with rimegepant were pain-free and 37% were free of their most bothersome symptoms at two hours compared to 14% and 28% that received placebo. In a 1,072 patient, Phase III trial, 20% of patients treated with rimegepant were pain-free and 38% were free of their most bothersome symptoms at two hours compared to 12% and 25% with placebo. Biohaven Pharmaceuticals is also developing an orally disintegrating tablet (ODT) formulation for rimegepant.
Stay current with drugs to treat or prevent migraines and other drugs in the late stages of development with the Prescribe Right Pharmaceutical Pipeline Tracker.
One new approval last week and some big news on two budget busters.
In a 507-patient, Phase III trial, Aveo Oncology’s tivozanib demonstrated improved progression-free survival, but not overall survival compared to sorafenib in patents with renal cancer. Tivozanib was similar to bevacizumab in colorectal cancer but did not show a benefit for breast cancer. Aveo announced that in a 27 patient, Phase Ib/II trial, data from 14 patients treated with tivozanib in combination with nivolumab suggested a partial response in 9 patients and 5 patients with disease stabilization. In a 161 patient, Phase III trial, advanced renal cell carcinoma patients that had been treated with sorafenib were changed to treatment with tivozanib and had a PFS of 11 months and overall survival of 22 months. Aveo announced that in the 351-patient, Phase III TIVO-3 trial, patients treated with tivozanib had a median progression-free survival of 5.6 months compared to 3.9 months with sorafenib in patients with highly refractory advanced or metastatic renal cell carcinoma that had failed at least two prior therapies, including a vascular EGFR TKI. A checkpoint inhibitor had been used in 26% of patients. Tivozanib has an FDA Orphan Drug priority designation.
The FDA has granted Fast Track designation to Karyopharm Therapeutics’ selinexor in addition to its Orphan Drug priority designation. Selinexor is intended to treat patients with diffuse large B-cell lymphoma. The drug has an April 6, 2019, PDUFA Date.
Mallinckrodt announced that in a 52-week, Phase II/III trial, VTS-270 was no better than placebo in treating Niemann-Pick type C. Mallinckrodt obtained VTS-270 when it bought Sucampo Pharmaceuticals in 2017.
Biomarin plans to file an NDA for valoctocogene roxaparvovec in the second half of 2019.
In a 58 patient, Phase II trial, 57% of patients treated with Jiangsu Chiatai Qingjiang/Advenchen’s anlotinib had a partial response and 85.5% of patients had a progression-free survival of 48 weeks in patients with advanced or metastatic medullary thyroid carcinoma.
The European Medicines Agency accepted the MAA for Ambit Biosciences Corporation/Daiichi Sankyo’s quizartinib and granted accelerated assessment for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) which is FLT3-ITD positive. The FDA has granted Orphan Drug, Breakthrough Therapy and Fast Track priority designations to quizartinib.
AveXis indicated it may price AVXS-101 at $4 million or more for the single dose treatment of spinal muscular atrophy.
Sage announced it will price brexanolone between $20,000 and $35,000 per course of treatment. At least one analyst estimates that rebates will bring the price down to $14,000. Due to the risk of dizziness and loss of consciousness, Sage indicated that brexanolone will likely be approved with a REMS program requiring administration and monitoring in a healthcare setting with REMS program certified staff. Sage forecasts availability in March 2019 and will initially focus its sales force on hospitals that are able to meet REMS requirements.
The FDA approved revefenacin inhalation solution (Yupelri, Mylan Pharmaceuticals, Theravance Biopharma) on 11/9/18 for the maintenance treatment of patients with COPD. Yupelri is the first muscarinic receptor antagonist that is available as a nebulized solution.
Stay current with drugs in the late stages of development with the Prescribe Right Pharmaceutical Pipeline Tracker.
Besting September’s approvals, the FDA approved six new drugs during October for the following uses:
Here’s the link to the full details:
During the first week of October 2018
The FDA approved Almirall’s and Paratek Pharmaceuticals’ sarecycline (Seysara) on 10/2/18, ahead of the PDUFA Date, for the treatment of patients aged 9 years and older with inflammatory lesions associated with non-nodular moderate to severe acne vulgaris. No Priority Designations.
The FDA approved Paratek Pharmaceutical’s omadacycline (Nuzyra) on 10/2/18, ahead of its PDUFA Date, for the treatment of adults with community-acquired bacterial pneumonia or acute skin and skin structure infection. Omadacycline is a once-daily IV and oral antibiotic. Pricing is expected to be similar to linezolid (Zyvox). The drug had Fast Track and Qualified Infectious Disease Product Priority Designations.
The FDA has approved Ionis Pharmaceuticals’ inotersen (Tegsedi) on 10/5/18, for the treatment of hereditary transthyretin-mediated amyloidosis polyneuropathy in adults. Inotersen is a once-weekly subcutaneous injection. Inotersen will be available through a REMS program with distribution through Accredo specialty pharmacy, a subsidiary of Express Scripts. The drug has a black box warning for thrombocytopenia and glomerulonephritis. Inotersen will be priced at $450,000. ICER concluded the drug would need to be priced at $96,103 to be cost-effective. The drug has Fast Track and Orphan Drug Priority Designations.
The FDA approved elapegademase (Revcovi , Leadiant Biosciences), a new enzyme replacement therapy, on 10/5/18 for the treatment of adenosine deaminase severe combined immune deficiency.
The FDA rejected ferric maltol during Shield’s first submission, but the company has resubmitted an NDA, which was accepted by the FDA this month.
The FDA granted a priority review for Karyopharm Therapeutics’ selinexor and assigned a PDUFA date of April 6, 2019.
The FDA rejectedAcacia Pharma’s amisulpride’s NDA on 10/8/18 due to manufacturing issues.
An FDA review of oliceridine was skeptical the drug had less respiratory depression than morphine and the highest dose (0.5mg) appeared to be less efficacious than morphine in analgesic effects, but similar in ADR. Lower doses had few ADR, but less analgesic effects. All strengths of olicerdine provided more analgesic effect than placebo. The FDA’s Anesthetic and Analgesic Drug Products Advisory Committee voted 8-7 against recommending approval of oliceridine.
In a review the FDA found sublingual sufentanil tablets to be efficacious for moderate-to-severe acute pain but had concerns over the maximum dosing and the risk of misplaced tablets due to their small size. AcelRx and the FDA have both proposed a REMS program for sublingual sufentanil tablets. The FDA’s Anesthetic and Analgesic Drug Products Advisory Committee voted 10-3 to recommend approval of sublingual sufentanil tablets.
The FDA approved Medivation’s talazoparib (Talzenna) on October 16, nearly a month ahead of its PDUFA Date, for the treatment of advanced BCRA-positive breast cancer.
An FDA Advisory Committee voted 10-0 to recommend prucalopride tablets for the treatment of adults with chronic idiopathic constipation. In a review of Shire and Janssen’s prucalopride, the FDA noted that despite the drug being available in Europe since 2009, there have been no controlled trials lasting at least 12-months. In another FDA review, an integrated safety analysis of 2552 patients the most common ADR in prucalopride trials were gastrointestinal disorders including nausea, diarrhea, abdominal pain, and headache with similar cardiovascular events compared to placebo. A final decision on prucalporide is expected in December. The drug does not have a PDUFA Date nor any Priority Designation.
Finally, in a slow last week of the month saw the FDA approved Genentech/Shionogi’s baloxavir marboxilon 10/24/18, two months ahead of its PDUFA Date, for the treatment of acute uncomplicated influenza in patients 12 year or older that have been symptomatic for 48 hours or less. There is only one other drug focused on Influenza in the pipeline.
Check out our subscription options, so you can stay up you date with those budget busters that are on their way.
The FDA approved lorlatinib (Lorbrena) on 11/2/18 for the treatment of patients with ALK-positive non-small cell lung cancer (NSCLC) who have progressed on crizotinib and at least one other ALK inhibitor.
The FDA approved sufentanil sublingual tablets on 11/2/18 for the treatment of moderate-to-severe acute pain in certified medically supervised healthcare settings.
The FDA's Anesthetic and Analgesic Drugs Advisory Committee voted 8-7 against recommending approval of oliceridine in October 2018. The FDA rejected oliceridine in November 2018 and requested additional data on QT prolongation, a larger safety database and more nonclinical data and validation reports.
The FDA’s Psychopharmacologic Drugs Advisory Committee and Drug Safety and Risk Management Committee voted 21-2 against recommending approval of Alkermes' buprenorphine/samidorphan in a joint meeting in November 2018. The committee felt that efficacy had not been proven and studies had methodological problems, but in a narrow vote (13-10) indicated that safety had been demonstrated.
The FDA noted a loss of consciousness during infusion of brexanolone in 6 patients during clinical trials and recommended a REMS program and administration by a health care professional outside the patient’s home. In current data the increased risk for loss of consciousness/presyncope is 4%. The FDA’s Psychopharmacologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee voted 17-1 this month that brexanolone demonstrated efficacy and 16-2 for safety in the treatment of postpartum depression.
The FDA granted Sanofi’s Dengvaxia dengue vaccine a priority review and set a PDUFA date of 5/1/2019.
Esperion announced results of a Phase III trial, where the addition of bempedoic acid to a statin resulted in MACE events of 6.1% compared to 8.2% with statin monotherapy in patients with atherosclerotic cardiovascular disease and/or heterozygous familial hypercholesterolemia. At 12 weeks, LDL was decreased an additional 18% with bempedoic acid compared to placebo. ADRs were similar with the addition of bempedoic acid to a statin compared to statin monotherapy.
Novartis announced results from two Phase III trials involving patients with AMD. Brolucizumab given every 3 months was non-inferior to aflibercept given every 2 months in best-corrected visual acuity at 48 weeks. Intra-retinal fluid and/or sub-retinal fluid was found in 24% of brolucizumab patients compared to 37% of aflibercept patients, while in HARRIER it was found in 24% of brolucizumab patients compared to 39% of aflibercept patients.
ViiV announced that in a Phase IIb Trial of cabotegravir and rilpivirine, 90% of patients that received the 8-week regimen and 83% of patients on the four-week regimen remained virally suppressed, while 97% and 100% of patents switched from an oral regimen remained virally suppressed. ViiV announced that in another Phase III trial, patients that were viral suppressed at 20 weeks with abacavir/dolutegravir/lamivudine remained suppressed with monthly injected cabotegravir and rilpivirine that was comparable in HIV viral suppression to continuation of abacavir/dolutegravir/lamivudine.
In a Phase II trial, patients treated with remimazolam for endoscopy sedation showed a success rate of 32%, 56%, and 64% for the low, medium and high dose groups of remimazolam compared to the 44% success rate with midazolam. A Phase IIb trial found a 92% success rate with remimazolam and 75% success rate with midazolam in sedation for colonoscopy. In a Phase III trial, successful sedation for colonoscopy was achieved in 91% of patients with remimazolam, 25% with midazolam and 2% with placebo. In a 446-patient trial, successful sedation for bronchoscopy was achieved in 80% of patients with remimazolam, 33% with midazolam and 5% with placebo.
GSK announced topline results from a Phase III trial, where oral daprodustat was non-inferior to darbepoetin in raising mean hemoglobin levels during weeks 40 to 52 in dialysis-dependent patients with anemia associated with chronic kidney disease.
In a Phase II, open label trial, tremelimumab added to durvalumab did not improve the objective response rate compared to durvalumab in patients with recurrent or metastatic head and neck squamous cell carcinoma.
In a Phase IIb, dose ranging trial, vibegron decreased average daily micturitions and the number of urge incontinence episodes compared to placebo in patients with overactive bladder. The combination of vibegron and tolterodine extended release improved daily micturitions and the number of urge incontinence episodes compared to monotherapy with either agent.
Astellas announced that in a Phase III trial, 57.7% of patients treated with peficitinib 100mg achieved AR20, 74.5% with 150mg and 30.7% with placebo in rheumatoid arthritis patients with an inadequate response to at least one conventional DMARD.
A phase III trial is underway for high risk locally advanced cervical cancer (NCT02853604). Advaxis’ axalimogene filolisbac is being studied in phase II trials for metastatic anal cancer, metastatic cervical head and neck cancer (in combination with durvalumab) and metastatic cervical cancer. The FDA has placed a hold on a Phase I/II trial examining the effect of axalimogene filolisbac plus durvalumab for the treatment of HPV-associated cervical or head and neck cancers due to a death during the trial. Advaxis discontinued the Phase I/II HPV-associated cervical or head and neck cancers trial and canceled a Phase III H cervical cancer trial. Advaxis withdrew its MAA for axalimogene filolisbac.
Stay current with drugs in the late stages of development with the Prescribe Right Pharmaceutical Pipeline Tracker.
As we reported in our weekly update on Tuesday, the FDA approved baloxavir marboxil (Xofluza from Roche and Shionogi for the treatment of acute uncomplicated influenza in patients 12 year or older that have been symptomatic for 48 hours or less. The approval came two months ahead of its 12/24/2018 PDUFA Date. The drug had no priority designation.
Here are the potential budget- busting investigational drugs with November PDUFA Dates - two are due by this weekend.:
Oliceridine from Trevena with a PDUFA Date of Nov 2, 2018 is an Opioid analgesic. It is delivered Intravenously Bolus for Post-op pain. An FDA advisory committee voted 8-7 not to recommend approval of oliceridine and recommended additional clinical trials. The committee also noted the potential for QT prolongation.
Sublingual sufentanil (Desuvia) from AcelRx Pharmaceuticals has a Nov 3, 2018, PDUFA Date. It’s an Opioid analgesic for Post-op and battlefield pain. The FDA’s Anesthetic and Analgesic Drug Products Advisory Committee voted 10-3 to recommend approval of sublingual sufentanil tablets.
Mid-month, revefenacin from Mylan Pharmaceuticals/Theravance Biopharma has a Nov 13, 2018, PDUFA Date. This drug is a muscarinic receptor antagonist for COPD that is administered by nebulization, offering an alternative for patients unable to use a MDI.
Secondly at mid-month, Rifamycin-SV from Salix/Cosmo Pharmaceuticals whose PDUFA Date is Nov 16, 2018. Rifamycin is a single dose oral antibacterial agent to treat Traveler's diarrhea. Rifamycin has Fast Track and Qualified Infectious Disease Product priority designations.
Finally, we have four end-of-the-month budget-busters:
Larotrectinib from Loxo Oncology/Bayer with a Nov 26, 2018, PDUFA Date. Larotrectinib targets tumors with NTRK-fusion proteins. Loxo estimates there are 1,500 and 5,000 late-stage cancer patients in the U.S. and a similar number in Europe with the mutation. In November 2017, Bayer bought global development rights for larotrectinib. The FDA has granted larotrectinib an accelerated review for use in treating locally advanced or metastatic tumors harboring a neurotrophic tyrosine receptor kinase (NTRK) gene fusion regardless of site.
Amifampridine (Firdapse) from Catalyst/BioMarin Pharmaceutical, PDUFA Date of Nov 28, 2018, is a Potassium Channel Blocker in oral form to treat Lambert-Eaton Myasthenic Syndrome (LEMS) and Spinal Muscular Atrophy (SMA). There is controversy over the drug because it is a slightly tweaked version of an old drug, 3,4-DAP, from U.S. drug maker Jacobus Pharmaceutical, who is giving it away for free. Concern is that Catalyst could charge $60,000 to $80,000 per year for providing little development or improvement. Amifampridine has Orphan Drug and Breakthrough Therapy priority designations.
Gilteritinib from Astellas/Kotobuk with a Nov 29, 2018, PDUFA Date, is an oral Tyrosine Kinase Inhibitor targeting Acute Myeloid Leukemia and Non-small cell lung cancer. The drug has orphan drug and fast track priority designations.
Pfizer’s lorlatinib closes the month with a Nov 30, 2018, PDUFA Date. The drug is an oral ALK/ROS1 tyrosine kinase inhibitor for Anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC). Loriatinib has Orphan Drug and Breakthrough Therapy priority designations.
Check out our subscription options, so you can stay current with drugs that will strain your budget.
Stay informed, subscribe to the
Prescribe Right Pharmaceutical Pipeline Tracker
Latest Tweets from Prescribe Right