===Establishment Labs (ESTA) : FDA approves Motiva breast implants

 

Establishment Labs receives approval from the U.S. FDA for the use of Motiva SmoothSilk Ergonomix and Motiva SmoothSilk Round breast implants in primary and revision breast augmentation

• The Motiva U.S. IDE Study was conducted at 32 centers in the U.S. and three centers in Western Europe. At the study endpoint of three years post-implantation, patient compliance in the primary augmentation cohort of the trial was 92.4%.
• The three-year, by-patient, Kaplan-Meier risk rates of first occurrence of complications for patients (95% confidence interval) in the primary augmentation cohort (N=451) were as follows: Capsular Contracture: 0.5%; Rupture, Confirmed or Suspected: 0.6% (MRI Cohort N=176); Breast Pain: 0.7%; Infection: 0.9%. Any reoperation, including an implant size change, was reported at 6.1% and any complication, including reoperations, was reported at 8.4%.

==BridgeBio Pharma (BBIO) : Breakthrough Therapy Designation to oral infigratinib

 

BridgeBio Pharma announces that the FDA has granted Breakthrough Therapy Designation to oral infigratinib under development for children with achondroplasia

• Breakthrough Therapy Designation was granted based on preliminary clinical evidence from the PROPEL 2 clinical trial, meeting the FDA's requirement of potentially demonstrating substantial improvement in efficacy over available therapies on clinically significant endpoint(s).
• BridgeBio will leverage the benefits of Breakthrough Therapy Designation to expedite the development and regulatory review of infigratinib in the United States and if approved, infigratinib could be the first-in-class oral therapeutic option for children living with achondroplasia.
• In Cohort 5 of PROPEL 2 (0.25 mg/kg/day), oral treatment with infigratinib resulted in a statistically significant and sustained increase in annualized height velocity (AHV), with a mean change from baseline of +2.51cm/yr at Month 12, and +2.50 cm/yr at Month 18 (p=0.0015) and statistically significant improvement in body proportionality at Month 18.

==Sarepta Therapeutics (SRPT) : expanded FDA approval of ELEVIDYS to Duchenne Muscular Dystrophy patients

 

Sarepta Therapeutics announces expanded FDA approval of ELEVIDYS to Duchenne Muscular Dystrophy patients

• FDA grants traditional approval to ELEVIDYS for ambulatory Duchenne patients. FDA grants accelerated approval to ELEVIDYS for non-ambulatory Duchenne patients.
• Consistent with the accelerated approval pathway, Sarepta has committed to conduct and submit the results of a randomized, controlled trial to verify and confirm the clinical benefit of ELEVIDYS in patients with Duchenne muscular dystrophy who are non-ambulatory. ENVISION (Study SRP-9001-303), a global, randomized, double-blind, placebo-controlled Phase 3 study of ELEVIDYS in non-ambulatory and older ambulatory individuals with Duchenne, is underway and intended to serve as this postmarketing requirement.

===Amylyx Pharmaceuticals (AMLX) announces FDA Advisory Committee supports approval of AMX0035 for the treatment of ALS

 

Amylyx Pharmaceuticals announces FDA Advisory Committee supports approval of AMX0035 for the treatment of ALS 

• Co announced the FDA Peripheral and Central Nervous System Drugs Advisory Committee (PCNSDAC) voted (7 yes votes and 2 no votes) that the available evidence of effectiveness is sufficient to support approval of AMX0035 (sodium phenylbutyrate and taurursodiol [also known as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis (ALS).
• The PCNSDAC's decision was based on a review of all available evidence, including new analyses submitted for discussion at the September 7 meeting and the information presented at the March 30, 2022, PCNS meeting.
• The PCNSDAC recommendations, while not binding, will be considered by the FDA in its review of the pending New Drug Application (NDA) for AMX0035. As previously reported, the Prescription Drug User Fee Act target action date for the NDA is September 29, 2022, which was extended by the FDA to allow more time to review additional analyses of data from the Company's clinical studies.

Novavax (NVAX) finally nabs Covid-19 vaccine EUA

a. 6/22:  vol 6.7M; 2, 4, 5, 10, 11, 52, 95

b.  7/1: vol 6.4M; 12, 23, 60, 67, 82, 83

 

 

 

 

Novavax confirms that its COVID-19 vaccine has received EUA from the FDA

• Co announces that the Novavax COVID-19 Vaccine, Adjuvanted (NVX-CoV2373) has received EUA from the FDA to provide a two-dose primary series for active immunization.
• "Today's FDA emergency use authorization of our COVID-19 vaccine provides the U.S. with access to the first protein-based COVID-19 vaccine."
• The FDA EUA was based on data from the pivotal Phase 3 clinical trial, PREVENT-19, which enrolled approximately 30,000 participants.

In the U.S., the fact sheet accompanying the vaccine warns of a heightened risk of inflammation of the heart and tissues surrounding it following administration. That's in line with the same sheets for Pfizer's (PFE) and Moderna's (MRNA) Covid vaccines. On Thursday, regulators in Europe added a warning against the risk of severe allergic reaction to the Novavax shot.

Still, experts hope adding a third vaccine technology to the U.S. coffers will help bolster the number of people vaccinated against Covid. Novavax's jab uses a protein-based technology and a tree bark chemical from Chile to boost the immune response. Pfizer and Moderna use newer messenger RNA platforms. Johnson & Johnson (JNJ) employs an empty virus to deliver its single-shot vaccine.

NVAX Stock: Small Portion Of Unvaccinated People

Novavax is late to the market, however. Pfizer and Moderna won FDA authorization in December 2020 and J&J followed in February 2021. Further, the company tested its vaccine before the emergence of the delta and omicron variants.

Novavax has global authorizations, including in younger people, but in the U.S. it will only be allowed in adults who have yet to receive a Covid shot. The Centers for Disease Control and Prevention estimates that group accounts for just 10% of people in the country.

-=Adamis Pharma (ADMP) : opioid overdose treatment gets FDA approval

 

Adamis Pharma announces that the U.S. Food and Drug Administration has approved Adamis' ZIMHI 5 mg/0.5 mL product

ZIMHI is a high-dose naloxone injection product FDA-approved for use in the treatment of opioid overdose.

=AcelRx Pharmaceuticals (ACRX) : opioid painkiller DSUVIA to be approved by FDA

• AcelRx confirms FDA Advisory Committee voted 10-3 in favor of recommending the approval of DSUVIA for the treatment of moderate-to-severe acute pain in adult patients

FDA staff had said the company's opioid painkiller was safe and effective for use based on resubmitted data. An advisory panel to the FDA will vote on the drug on Friday, before the regulator’s final decision by Nov 3. The staff reviewers are still concerned over the risk of accidental exposure due to misplaced tablets. The FDA's Anesthetic and Analgesic Drug Products Advisory Committee is evaluating the company's new drug application for Dsuvia. The committee said the dosage regimen appear effective in the drug's proposed population and reasonably safe in the context of existing opioid therapy.

=Portola Pharma (PTLA) receives FDA approval for Andexxa

Portola Pharma receives FDA approval for Andexxa  

• Co announced that the FDA has approved Andexxa, the first and only antidote indicated for patients treated with rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.
• Andexxa received both U.S. Orphan Drug and FDA Breakthrough Therapy designations and was approved under the FDA's Accelerated Approval pathway based on the change from baseline in anti-Factor Xa activity in healthy volunteers. Continued approval for this indication may be contingent upon post-marketing study results to demonstrate an improvement in hemostasis in patients.

Insys Therapeutics (INSY): granted Fast Track designation

• Insys Therapeutics: FDA has granted Fast Track designation to the company's cannabidio oral solution for the treatment of Prader-Willi syndrome; co plans to start the clinical development program in late first quarter of 2018

 

=Alexion Pharma (ALXN) receives FDA approval for Soliris

Alexion Pharma receives FDA approval for Soliris 

• Co announced the FDA approved Soliris (eculizumab) as a treatment for adult patients with generalized myasthenia gravis who are anti-acetylcholine receptor antibody-positive.
• In the Phase 3 REGAIN study and its ongoing open-label extension study, Soliris demonstrated treatment benefits for patients with anti-AchR antibody-positive gMG who had previously failed immunosuppressive treatment and continued to suffer from significant unresolved disease symptoms, which can include difficulties seeing, walking, talking, swallowing and breathing. 
• These patients are at an increased risk of disease exacerbations and crises that may require hospitalization and intensive care and may be life-threatening.
• These patients represent approximately 5-10% of all patients with MG.

Mylan N.V. (MYL) receives FDA approval for Copaxone 40 mg /ml generic

  

Mylan N.V. receives FDA approval for Copaxone 40 mg /ml generic

• Co announced the FDA has approved Mylan's Abbreviated New Drug Applications for Glatiramer Acetate Injection 40 mg/mL for 3-times-a-week injection, an AP-rated, substitutable generic version of Teva's Copaxone 40 mg/mL, and Glatiramer Acetate Injection 20 mg/mL for once-daily injection, an AP-rated, substitutable generic version of Teva's Copaxone 20 mg/mL, which are indicated for the treatment of patients with relapsing forms of multiple sclerosis, a chronic inflammatory disease of the central nervous system. Mylan will begin shipping imminently.
• The FDA approved Mylan's Glatiramer Acetate Injection 40 mg/mL for 3-times-a-week injection and 20 mg/mL for once-daily injection as therapeutic AP-rated equivalents to Copaxone 40 mg/mL and Copaxone 20 mg/mL, respectively, meaning if they are substituted for their branded counterpart they can be expected to have the same clinical effect and safety profile. As part of its ANDAs, Mylan submitted rigorous side-by-side analyses, including characterization data, which demonstrated that Mylan's Glatiramer Acetate Injection 20 mg/mL and 40 mg/mL have the same active ingredient, dosage form, route of administration and strength as their branded counterpart. Mylan's Glatiramer Acetate Injection 20 mg/mL and 40 mg/mL are available by prescription only.

=The Medicines Co (MDCO) : FDA approves Vabomere

The Medicines Co confirms FDA approval of VABOMERE for injection for the treatment of adult patients with complicated urinary tract infections
The co expects that VABOMERE will be available in the fourth quarter of 2017. The FDA approval of VABOMERE triggered a $40 million milestone payment obligation to the former securityholders of Rempex Pharmaceuticals, Inc., which the co acquired in December 2013.

Impax Laboratories (IPXL) : FDA approves a generic version of Concerta

• Update: In 2018, Impax Laboratories merged with Amneal Pharmaceuticals LLC, to form Amneal Pharmaceuticals, Inc. Shares of newly public AMRX began trading on the NYSE on May 7, 2018.

 

*****

Concerta (Methylphenidate)  

• Stimulant
• Brands: Concerta, Daytrana, Methylin, Ritalin, and Aptensio
• It can treat ADHD and narcolepsy.

 

Impax Labs announces AB therapeutic equivalent rating and final FDA approval on its Abbreviated New Drug Application for a generic version of Concerta :

Co has received an AB therapeutic equivalent rating and final U.S. Food and Drug Administration ("FDA") approval on its Abbreviated New Drug Application ("ANDA") for a generic version of Concerta (JNJ) Extended-Release tablets USP CII, 18, 27, 36 and 54 mg.

"We are preparing for launch including working to secure API quota and currently expect to launch by the end of this year. As a result, we don't anticipate sales of generic Concerta to meaningfully impact our earnings in 2017."

Methylphenidate hydrochloride extended-release tablets had U.S sales of ~$1.8 billion, according to IMS Health for the 12 months ending May 2017.

Arena Pharmaceuticals (ARNA) before FDA decision on Wed 6/27/12

Arena Pharmaceuticals (ARNA) shares tumbled Friday, and activity in options shot higher, ahead of a key ruling from the U.S. Food and Drug Administration on the company's weight-loss drug.

  

Options watchers said stock holders were moving into options as a way to play the FDA ruling while limiting risk. A decision is expected Wednesday on the company's weight-loss drug candidate lorcaserin. If given the go-ahead, lorcaserin would be the first prescription diet drug to receive U.S. approval in more than a decade.

A lot of the downward pressure on the stock appeared to come from investors taking profits and moving into options ahead of the decision, according to William Lefkowitz, options strategist at vFinance.

"It looks like investors are taking profit, but then rolling some of that profit into 'call' options," said Mr. Lefkowitz. "They still get to participate if the news is really good next week, but they get to limit their losses to what they put into the options."

Call options grant the right to buy stock at a set price by a designated date. When an investor buys a call option, the risk is limited to the cost of that option. Even if the shares fall sharply, the trader's maximum loss remains the price paid for the option.

Arena shares dropped $1.80, or 15%, to $9.88 Friday after the shares had more than quintupled in value leading up to the decision. Just three months ago, the shares traded at $2.

Activity in call options was strong across the board, with options expiring in July busy at the $10 through $15 strike prices. Meanwhile, weekly options expiring next Friday were also active, with $12 and $15 call options seeing the most action.

Yet not all option buyers were as optimistic ahead of the advisory-panel decision.

One trader bought more than 9,300 July $8 put options, which grant the right to sell shares at that price, for $1.61 a piece. That means the stock must drop at least 36% over the next four weeks for the bet to break even.

Arena has been unprofitable every quarter since March 2001, according to FactSet data.

Options activity in the San Diego pharmaceutical company soared to more than four times the daily average Friday. The high price of option contracts suggests traders are pricing in a nearly 50% swing one way or the other in the wake of the FDA decision.

*** 2 days after the FDA approval ***
all gains (400%) happened in the few months leading to the decision