US FDA approval tracker: February 2023

 There were several high-profile approvals last month, including for Apellis’s Syfovre, given a broad label and a flexible dosing regimen in geographic atrophy. However, Apellis might not have the market to itself for long as Iveric Bio’s Zimura has a Pdufa date in August. Reata gained a surprise greenlight for its Friedreich's ataxia therapy Skyclarys, despite a complex regulatory path. However, Cytokinetics received a knockback for its heart failure project omecamtiv after an earlier negative adcom. The company has no plans to run another study, leaving it to focus on aficamten in obstructive hypertrophic cardiomyopathy, with phase 3 data due later this year. Lastly, the outcome of an adcom for Pfizer’s adult respiratory syncytial virus (RSV) vaccine Abrysvo was less clear-cut than might have been expected, with a 7 to 4 vote in favour. Panellists highlighted incidences of Guillain-Barré Syndrome, and post-marketing studies are expected to evaluate this risk. Stifel analysts note that GSK, whose own vaccine Arexvy is up before a panel today, has agreed with the FDA to monitor the syndrome as part of its pharmacovigilance programme.

Notable first-time US approval decisions in February
Project	Company	Indication(s)	2028e SBI ($m)	Outcome
Syfovre (intravitreal pegcetacoplan)	Apellis	Geographic atrophy secondary to age-related macular degeneration	2,310	Approved
Altuviiio (efanesoctocog alfa)	Sanofi	Ultra-long-acting FVIII therapy for haemophilia A	879	Approved
Skyclarys (omaveloxolone)	Reata	Friedreich's ataxia in patients aged 16 and older	838	Approved
Fezolinetant	Astellas	Moderate to severe vasomotor symptoms associated with menopause	699	Extended to 22 May (more time to review)
Filspari (sparsentan)	Travere	IgA nephropathy	412	Approved (accelerated)
Jesduvroq (Duvroq, daprodustat)	GSK/Kyowa Kirin	Anaemia due to CKD in adults on/not on dialysis	355	Approved in dialysis patients, CRL in non-dialysis
Omecamtiv mecarbil	Cytokinetics	Reduce the risk of CV death and heart failure events in patients with symptomatic chronic HFrEF	326	CRL (additional trial required)
Lamzede (velmanase alfa)	Chiesi	Alpha-mannosidosis	-	Approved
SBI: sales by indication. Source: Evaluate Pharma & company releases.

 

 

Advisory committee meetings in February
Project	Company	Indication	2028e SBI ($m)	Outcome
Abrysvo (RSVpreF)	Pfizer	Prevention of acute respiratory disease and lower respiratory tract disease caused by RSV in adults ≥60 years	1,216	7 to 4 in favour (note GSK's vaccine Arexvy has an adcom on March 1)
Narcan nasal spray (4mg/0.1ml)	Emergent Biosolutions	Nonprescription treatment of known or suspected opioid overdose	175	Unanimously in favour
Jemperli	GSK	Monotherapy in early treatment of MMRd/MSI-high rectal cancer	-	8 to 5 in favour of data from two single-arm trials being sufficient to characterise benefits and risks (Jemperli moves towards adjuvant rectal cancer at last)
RSV: respiratory syncytial virus. Source: Evaluate Pharma, company releases & FDA adcom calendar.

 

Supplementary and other notable approval decisions in February
Product	Company	Indication (clinical trial)	Outcome
Empaveli	Apellis	PNH, treatment-naive patients, and long-term safety and efficacy data (Prince, Pegasus)	Approved
Trodelvy	Gilead	Pretreated HR+/Her2-metastatic breast cancer (Tropics-02)	Approved
Eylea	Regeneron/ Sanofi	Retinopathy of prematurity in preterm infants (Firefleye, Butterfleye)	Approved
Tezspire	Astrazeneca/ Amgen	Self-admin in a pre-filled, single-use pen for patients ≥12 years with severe asthma (Path-Home, Path-Bridge)	Approved
Kevzara	Sanofi/ Regeneron	Polymyalgia rheumatica (Saphyr)	Approved
Takhzyro	Takeda	Hereditary angioedema attacks in children 2 to <6 years (Help, Spring)	Approved
Jemperli	GSK	MMRd endometrial cancer, progressed on or following a prior platinum-containing regimen (long-term outcomes from Garnet)	Full approval
Cibinqo	Pfizer	Adolescents with refractory, moderate-to-severe atopic dermatitis whose disease is not adequately controlled with other systemic drug products (Jade Teen)	Approved
Austedo XR	Teva	Once-daily formulation for tardive dyskinesia and chorea associated with Huntington’s disease	Approved
Voquezna (Vonaprazan, Takecab)	Phathom	Erosive oesophagitis (Phalcon-ee) and H pylori	CRLs, stability data requested owing to impurity previously found in product, launch of Voquezna Dual and Triple Pak on hold (H pylori indication)
Krabeva/MYL-1402O (Avastin biosimilar)	Biocon	Certain oncology indications	CRL (facility inspection issues)
Source: Evaluate Pharma & company releases. https://www.evaluate.com/vantage/articles/insights/nme-approvals-snippets/us-fda-approval-tracker-february-2023

Organogenesis: Positive Interim Phase 3 Clinical Trial of ReNu for Knee Osteoarthritis

 Organogenesis Holdings Inc. (Nasdaq: ORGO), a leading regenerative medicine company focused on the development, manufacture, and commercialization of product solutions for the Advanced Wound Care and Surgical and Sports Medicine markets, today reported the favorable outcome of the interim analysis of its Phase 3 clinical trial for the management of symptoms associated with knee osteoarthritis.

The pre-specified interim analysis on 50% of the 474 required patients with moderate to severe knee osteoarthritis (Kellgren-Lawrence [KL] severity grade 2 to grade 4) focused on the 6-month primary endpoint for potential sample size re-estimation. The Independent Data Monitoring Committee (DMC) for the trial provided directional guidance on the results of the interim analysis, while rigorously maintaining all aspects of study blinding. The DMC recommended that the trial proceed without modification and without increase to sample size. Additionally, the DMC found the safety data to be consistent with the known safety profile for ReNu.

https://finance.yahoo.com/news/organogenesis-reports-positive-interim-analysis-210100687.html

Pivotal Phase 3 Study of Ambrx Treatment of HER2 Positive Metastatic Breast Cancer Achieves Positive Results

 Interim analysis of the ACE-Breast-02 study being conducted by Ambrx’s partner, NovoCodex Biopharmaceuticals, was completed by the Independent Data Monitoring Committee (IDMC)

The pivotal Phase 3 ACE-Breast-02 study met its pre-specified interim primary efficacy endpoint with statistical significance, demonstrating a greater progression free survival benefit compared to the control

Ambrx Biopharma Inc., or Ambrx, (NYSE: AMAM) announced today that it has been informed by its partner, NovoCodex Biopharmaceuticals, Inc. (NovoCodex), that an interim analysis for ACE-Breast-02, a randomized Phase 3 breast cancer clinical trial investigating Ambrx’s ARX788, an anti-HER2 antibody drug conjugate (ADC), has met its pre-specified interim primary efficacy endpoint with statistical significance, demonstrating a greater progression free survival (PFS) benefit compared to the active control.

ACE-Breast-02 is a randomized, controlled pivotal Phase 3 clinical trial of humanized anti-HER2 monoclonal antibody-AS269 conjugate (ARX788) in the treatment of HER2 positive patients with locally advanced or metastatic breast cancers in China.

The trial enrolled 441 HER2 positive breast cancer patients who have been previously treated with taxane and trastuzumab. Eligible subjects were randomly assigned in a 1:1 ratio to receive ARX788 or the control drugs, lapatinib combined with capecitabine. The primary endpoint was PFS based on Blinded Independent Review Committee (BIRC) assessment. An interim analysis was conducted by an Independent Data Monitoring Committee (IDMC) when two thirds (2/3) of the PFS events occurred.

After reviewing the interim results, the IDMC informed NovoCodex that the ACE-Breast-02 study met its pre-specified interim primary efficacy endpoint with statistical significance, demonstrating a greater PFS benefit compared to the control.

Based on the results from the interim analysis, NovoCodex plans to submit a communication application to seek marketing approval in China pending discussion with National Medical Products Administration (NMPA).

https://finance.yahoo.com/news/ace-breast-02-pivotal-phase-130000772.html

Lilly Cuts Insulin Prices by 70% and Caps Patient Insulin Out-of-Pocket Costs at $35 Per Month

 Eli Lilly and Company (NYSE: LLY) today announced price reductions of 70% for its most commonly prescribed insulins and an expansion of its Insulin Value Program that caps patient out-of-pocket costs at $35 or less per month. Lilly is taking these actions to make it easier to access Lilly insulin and help Americans who may have difficulty navigating a complex healthcare system that may keep them from getting affordable insulin.

Today, Lilly is reducing the list price of insulins by:

• Cutting the list price of its non-branded insulin, Insulin Lispro Injection 100 units/mL, to $25 a vial. Effective May 1, 2023, it will be the lowest list-priced mealtime insulin available, and less than the price of a Humalog® vial in 1999.
• Cutting the list price of Humalog® (insulin lispro injection) 100 units/mL1, Lilly's most commonly prescribed insulin, and Humulin® (insulin human) injection 100 units/mL2 by 70%, effective in Q4 2023.
• Launching RezvoglarTM (insulin glargine-aglr) injection, a basal insulin that is biosimilar to, and interchangeable with, Lantus® (insulin glargine) injection, for $92 per five pack of KwikPens®, a 78% discount to Lantus, effective April 1, 2023.

"While the current healthcare system provides access to insulin for most people with diabetes, it still does not provide affordable insulin for everyone and that needs to change," said David A. Ricks, Lilly's Chair and CEO. "The aggressive price cuts we're announcing today should make a real difference for Americans with diabetes. Because these price cuts will take time for the insurance and pharmacy system to implement, we are taking the additional step to immediately cap out-of-pocket costs for patients who use Lilly insulin and are not covered by the recent Medicare Part D cap."

In addition to reducing the list price of its insulins, Lilly is making it easier for more people with diabetes to get Lilly insulins:   

• Effective immediately, Lilly will automatically cap out-of-pocket costs at $35 at participating retail pharmacies for people with commercial insurance using Lilly insulin.3
• People who don't have insurance can continue to go to InsulinAffordability.com and immediately download the Lilly Insulin Value Program savings card to receive Lilly insulins for $35 per month.

"We are driving for change in repricing older insulins, but we know that 7 out of 10 Americans don't use Lilly insulin. We are calling on policymakers, employers and others to join us in making insulin more affordable," continued Ricks. "For the past century, Lilly has focused on inventing new and improved insulins and other medicines that address the impact of diabetes and improve patient outcomes. Our work to discover new and better treatments is far from over. We won't stop until all people with diabetes are in control of their disease and can get the insulin they need."

Today's announcement builds on years of efforts by Lilly to close the gaps in the U.S. healthcare system that keep some people with diabetes from accessing affordable insulin. In recent years, Lilly has introduced multiple insulin affordability solutions that have made a real impact, including launching low-list-price, non-branded insulins in 2019, implementing the Lilly Insulin Value Program in 2020 and committing all of our insulins to the Medicare Part D Senior Savings Model in 2021. Because of these efforts, the average out-of-pocket cost for Lilly insulins has dropped to $21.80 over the last five years.

In the coming weeks, Lilly will launch a nationwide public awareness campaign to help ensure all Lilly insulin users understand how to access the company's industry-leading affordability solutions. For more information, go to Lilly.com/insulin.

https://www.morningstar.com/news/pr-newswire/20230301de28143/lilly-cuts-insulin-prices-by-70-and-caps-patient-insulin-out-of-pocket-costs-at-35-per-month

Ocugen upped to Buy from Neutral by Chardan

 Target $3.50

https://finviz.com/quote.ashx?t=OCGN&ty=c&ta=1&p=d

ANI started at Buy by Guggenheim

 Target $55

https://finviz.com/quote.ashx?t=ANIP&ty=c&ta=1&p=d

Travere started at Buy by Guggenheim

 Target $37

https://finviz.com/quote.ashx?t=TVTX&ty=c&ta=1&p=d