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Wadia doc helps 40 kids with genetic disease get 1cr/yr US drug free for life
Wadia doc helps 40 kids with genetic disease get 1cr/yr US drug free for life

Time of India

time2 days ago

  • Health
  • Time of India

Wadia doc helps 40 kids with genetic disease get 1cr/yr US drug free for life

Mumbai: Early Monday morning, 14-year-old Bhavin Khare and his mother, Tanuja, set off from Thane's Murbad for Wadia Hospital, Parel, around 100km away, with a sense of anticipation. They had to collect a US drug—estimated to cost over Rs 1 crore for a year's supply—that would help him control the debilitating symptoms of cystic fibrosis, a genetic disease he suffers from. "Bhavin has been struggling all his life. He manages to go to school only 50% of the days due to repeated cough and chest infections," Tanuja said, hoping the new drug would let him lead a "normal life". He is one of five children with cystic fibrosis who received a three-month supply of Trikafta, manufactured by US-based Vertex Pharmaceuticals, at the hospital Monday. Thirty-five others are scheduled to get it in the coming days. You Can Also Check: Mumbai AQI | Weather in Mumbai | Bank Holidays in Mumbai | Public Holidays in Mumbai "We have signed an MoU with the company which, on compassionate grounds, will provide a lifelong supply of Trikafta to 40 patients," said pulmonology department head Dr Parmarth Chandane, who communicated with the US company for almost a year. Due to patent laws, the drug isn't available in India and can only be imported. Affected children in wealthy nations have had access to the drug for almost eight years, and parents' groups in Karnataka, South Africa and Brazil have moved local courts seeking some mechanism like compulsory licensing to ensure their children get access to the drug, too. by Taboola by Taboola Sponsored Links Sponsored Links Promoted Links Promoted Links You May Like Libas Purple Days Sale Libas Undo "The active ingredients for the three-drug combination (Trikafta—elexacaftor/tezacaftor/ivacaftor) that is now being called a miracle, are made in India, but our children don't have access to the finished product," said a doctor who didn't want to be identified. The Wadia Hospital distribution is the beginning of an alternative access for Indian patients. "The drug has shown promise, with certain research reports claiming patients will be able to enjoy a normal lifespan of 80 years as against the 15-20 years one normally associates with the disease," said Dr Chandane, who communicated with the US company for almost a year, seeking access for the 80 patients registered at Wadia Hospital. The company, however, said it would only provide drugs for patients over six years of age, resulting in only 40 qualifying for the free-for-life supply. Each of the affected children has to take three drugs—two in the morning and one in the evening—daily to prevent the buildup of thick, sticky mucus that usually clogs organs such as the lungs, pancreas and intestines, which could further lead to malnutrition, poor growth, frequent respiratory infections, as well as chronic lung disease. It's learnt that Vertex Pharmaceuticals will provide lifelong supply of the drug to patients registered at CMC Vellore in Tamil Nadu, too. "We are also in talks with the company for our patients," said paediatrician Dr Indu Khosla from SRCC Hospital, Haji Ali. "By offering such costly and essential medication free of charge, we're removing financial barriers for families. These children are getting a chance to breathe easier, grow healthy and live longer," said Dr Minnie Bodhanwala, CEO of Bai Jerbai Wadia Hospital for Children.

Vertex Pharmaceuticals (VRTX) Reports Next Week: Wall Street Expects Earnings Growth
Vertex Pharmaceuticals (VRTX) Reports Next Week: Wall Street Expects Earnings Growth

Yahoo

time2 days ago

  • Business
  • Yahoo

Vertex Pharmaceuticals (VRTX) Reports Next Week: Wall Street Expects Earnings Growth

The market expects Vertex Pharmaceuticals (VRTX) to deliver a year-over-year increase in earnings on higher revenues when it reports results for the quarter ended June 2025. This widely-known consensus outlook is important in assessing the company's earnings picture, but a powerful factor that might influence its near-term stock price is how the actual results compare to these estimates. The stock might move higher if these key numbers top expectations in the upcoming earnings report, which is expected to be released on August 4. On the other hand, if they miss, the stock may move lower. While the sustainability of the immediate price change and future earnings expectations will mostly depend on management's discussion of business conditions on the earnings call, it's worth handicapping the probability of a positive EPS surprise. Zacks Consensus Estimate This drugmaker is expected to post quarterly earnings of $4.24 per share in its upcoming report, which represents a year-over-year change of +133.1%. Revenues are expected to be $2.89 billion, up 9.2% from the year-ago quarter. Estimate Revisions Trend The consensus EPS estimate for the quarter has been revised 0.31% higher over the last 30 days to the current level. This is essentially a reflection of how the covering analysts have collectively reassessed their initial estimates over this period. Investors should keep in mind that an aggregate change may not always reflect the direction of estimate revisions by each of the covering analysts. Price, Consensus and EPS Surprise Earnings Whisper Estimate revisions ahead of a company's earnings release offer clues to the business conditions for the period whose results are coming out. Our proprietary surprise prediction model -- the Zacks Earnings ESP (Expected Surprise Prediction) -- has this insight at its core. The Zacks Earnings ESP compares the Most Accurate Estimate to the Zacks Consensus Estimate for the quarter; the Most Accurate Estimate is a more recent version of the Zacks Consensus EPS estimate. The idea here is that analysts revising their estimates right before an earnings release have the latest information, which could potentially be more accurate than what they and others contributing to the consensus had predicted earlier. Thus, a positive or negative Earnings ESP reading theoretically indicates the likely deviation of the actual earnings from the consensus estimate. However, the model's predictive power is significant for positive ESP readings only. A positive Earnings ESP is a strong predictor of an earnings beat, particularly when combined with a Zacks Rank #1 (Strong Buy), 2 (Buy) or 3 (Hold). Our research shows that stocks with this combination produce a positive surprise nearly 70% of the time, and a solid Zacks Rank actually increases the predictive power of Earnings ESP. Please note that a negative Earnings ESP reading is not indicative of an earnings miss. Our research shows that it is difficult to predict an earnings beat with any degree of confidence for stocks with negative Earnings ESP readings and/or Zacks Rank of 4 (Sell) or 5 (Strong Sell). How Have the Numbers Shaped Up for Vertex? For Vertex, the Most Accurate Estimate is higher than the Zacks Consensus Estimate, suggesting that analysts have recently become bullish on the company's earnings prospects. This has resulted in an Earnings ESP of +1.82%. On the other hand, the stock currently carries a Zacks Rank of #3. So, this combination indicates that Vertex will most likely beat the consensus EPS estimate. Does Earnings Surprise History Hold Any Clue? While calculating estimates for a company's future earnings, analysts often consider to what extent it has been able to match past consensus estimates. So, it's worth taking a look at the surprise history for gauging its influence on the upcoming number. For the last reported quarter, it was expected that Vertex would post earnings of $4.22 per share when it actually produced earnings of $4.06, delivering a surprise of -3.79%. Over the last four quarters, the company has beaten consensus EPS estimates just once. Bottom Line An earnings beat or miss may not be the sole basis for a stock moving higher or lower. Many stocks end up losing ground despite an earnings beat due to other factors that disappoint investors. Similarly, unforeseen catalysts help a number of stocks gain despite an earnings miss. That said, betting on stocks that are expected to beat earnings expectations does increase the odds of success. This is why it's worth checking a company's Earnings ESP and Zacks Rank ahead of its quarterly release. Make sure to utilize our Earnings ESP Filter to uncover the best stocks to buy or sell before they've reported. Vertex appears a compelling earnings-beat candidate. However, investors should pay attention to other factors too for betting on this stock or staying away from it ahead of its earnings release. Expected Results of an Industry Player Regeneron (REGN), another stock in the Zacks Medical - Biomedical and Genetics industry, is expected to report earnings per share of $8.03 for the quarter ended June 2025. This estimate points to a year-over-year change of -30.5%. Revenues for the quarter are expected to be $3.34 billion, down 5.7% from the year-ago quarter. Over the last 30 days, the consensus EPS estimate for Regeneron has been revised 6.5% down to the current level. Nevertheless, the company now has an Earnings ESP of +8.76%, reflecting a higher Most Accurate Estimate. When combined with a Zacks Rank of #4 (Sell), this Earnings ESP makes it difficult to conclusively predict that Regeneron will beat the consensus EPS estimate. Over the last four quarters, the company surpassed consensus EPS estimates three times. Stay on top of upcoming earnings announcements with the Zacks Earnings Calendar. Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report Vertex Pharmaceuticals Incorporated (VRTX) : Free Stock Analysis Report Regeneron Pharmaceuticals, Inc. (REGN) : Free Stock Analysis Report This article originally published on Zacks Investment Research ( Zacks Investment Research Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data

The 3 Things That Matter for Vertex Pharmaceuticals Now
The 3 Things That Matter for Vertex Pharmaceuticals Now

Yahoo

time2 days ago

  • Business
  • Yahoo

The 3 Things That Matter for Vertex Pharmaceuticals Now

Key Points Vertex Pharmaceuticals' leading cystic fibrosis franchise still looks promising. It also has a couple of exciting newer launches, and could land another one. Shares of the large drugmaker could still deliver market-beating returns. 10 stocks we like better than Vertex Pharmaceuticals › Vertex Pharmaceuticals (NASDAQ: VRTX) has been a terrific stock to own over the past decade. The stock has outperformed the market over this period, thanks to consistent financial results and excellent innovative capabilities. But how much longer can the biotech keep things up? If you're interested in owning the stock for the long term, it's important to pay close attention to several parts of the company's business. Below are three key things to watch out for now. 1. Vertex's cystic fibrosis franchise Vertex is best known for its work in developing and marketing medicines that target cystic fibrosis (CF) at its genetic roots. CF is a rare disease affecting the lungs. Although many have attempted to challenge the biotech in this area, none have yet succeeded; Vertex's therapies remain the only option for CF patients. The company continues to generate substantial revenue from its CF franchise, and several developments are worth paying attention to now. In December Vertex earned approval in the U.S. for Alyftrek, a next-generation CF therapy with a more convenient once-daily dosing. This drug may soon become Vertex's most important product. The company is also seeking to develop newer medicines for CF patients who aren't eligible for any of its current treatments. VX-522, an investigational drug, could target many of these patients. Approximately 75% of the 94,000 people with CF in North America, Europe, and Australia are currently benefiting from Vertex's products, whereas only 33% in other regions are, so there's still room to grow. Keep a watchful eye on continued clinical and commercial progress within Vertex's most important therapeutic area. 2. How will newer launches perform? Vertex's success over the past decade has been driven by its CF franchise; it's had little diversification. However, the company recently earned approval for some medicines outside of its core market. In late 2023, Casgevy, a gene-editing therapy for two blood-related diseases that Vertex developed with CRISPR Therapeutics, landed regulatory approval. And in January of this year, Vertex's treatment for acute pain, Journavx, also got the green light from regulators in the U.S. Casgevy isn't yet contributing much to the biotech's results; gene-editing therapies are complex to administer. However, this one holds considerable potential, as there's little competition to speak of. At $2.2 million per treatment course in the U.S., and with roughly 60,000 patients in the regions Vertex is targeting, Casgevy should exceed blockbuster status. Pay close attention to Vertex's comments on Casgevy's ongoing commercial progress. The same applies to Journavx. Vertex developed this therapy to fill the need for non-opioid painkillers. It became the first oral pain-signal inhibitor approved by the U.S. Food and Drug Administration. Furthermore, Vertex is likely to seek label expansions for the medicine, including the treatment of painful diabetic peripheral neuropathy. 3. Vertex's new gem, in type 1 diabetes Vertex's next brand-new launch could be a medicine for type 1 diabetes (T1D) called zimislecel. The therapy is undergoing a phase 1/2/3 study, where it's showing real promise. For instance, most (10 out of 12) patients treated with zimislecel were insulin-free after one year of follow-up in the phase 1/2 portion of this clinical trial. All patients were also free of severe hypoglycemic events 90 days after treatment. People with T1D typically cannot produce insulin, but this therapy helps restore their ability to do so. It could work as a functional cure for the chronic disease. Vertex plans to request approval from regulatory authorities for zimislecel next year, provided there are no significant clinical setbacks. It would be a great addition to the company's lineup -- and another product for Vertex investors to monitor closely. Is the stock a buy? Vertex shares look attractive for several reasons. First, the company's dominance in CF still affords it ample room to grow revenue and profits at a steady pace for the foreseeable future. Second, although it's possible that another biotech will eventually crack the code and launch a competing CF therapy, Vertex is well prepared. That's precisely why the company has diversified its lineup in recent years, and will hopefully continue to do so with the launch of zimislecel. Third, Vertex still has an exciting pipeline in CF and elsewhere. For instance, the company's late-stage candidate for APOL-1-mediated kidney disease, inaxaplin, looks promising. Currently, there are no approved treatments that target the underlying causes of this condition. Vertex could be the first to launch one. Even if that program doesn't pan out, though, Vertex Pharmaceuticals' lineup, pipeline, and consistent financial results make its prospects attractive. Should you invest $1,000 in Vertex Pharmaceuticals right now? Before you buy stock in Vertex Pharmaceuticals, consider this: The Motley Fool Stock Advisor analyst team just identified what they believe are the for investors to buy now… and Vertex Pharmaceuticals wasn't one of them. The 10 stocks that made the cut could produce monster returns in the coming years. Consider when Netflix made this list on December 17, 2004... if you invested $1,000 at the time of our recommendation, you'd have $636,628!* Or when Nvidia made this list on April 15, 2005... if you invested $1,000 at the time of our recommendation, you'd have $1,063,471!* Now, it's worth noting Stock Advisor's total average return is 1,041% — a market-crushing outperformance compared to 183% for the S&P 500. Don't miss out on the latest top 10 list, available when you join Stock Advisor. See the 10 stocks » *Stock Advisor returns as of July 21, 2025 Prosper Junior Bakiny has positions in Vertex Pharmaceuticals. The Motley Fool has positions in and recommends CRISPR Therapeutics and Vertex Pharmaceuticals. The Motley Fool has a disclosure policy. The 3 Things That Matter for Vertex Pharmaceuticals Now was originally published by The Motley Fool Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data

Vertex Pharmaceuticals Incorporated (VRTX) Launches JOURNAVX, First Non-Opioid Acute Pain Treatment
Vertex Pharmaceuticals Incorporated (VRTX) Launches JOURNAVX, First Non-Opioid Acute Pain Treatment

Yahoo

time4 days ago

  • Business
  • Yahoo

Vertex Pharmaceuticals Incorporated (VRTX) Launches JOURNAVX, First Non-Opioid Acute Pain Treatment

We recently compiled a list of Vertex Pharmaceuticals Incorporated stands second on our list and has recently launched the first non opioid acute pain treatment. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX), a global biotech firm based in Boston, is known for developing innovative treatments for serious diseases like cystic fibrosis, sickle cell disease, and beta thalassemia. The company is recognized for its strong pipeline targeting unmet medical needs. In July 2025, Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) launched JOURNAVX (suzetrigine), a first-in-class, non-opioid treatment for moderate-to-severe acute pain. JOURNAVX, an oral NaV1.8 pain signal inhibitor, was named a 2025 Breakthroughs Innovation Celebration winner by Premier, Inc. It represents the first new class of pain medicine in decades and offers a safer alternative to opioids by directly targeting pain pathways without addiction risks. This launch marks a major advancement in pain management, aligning with public health efforts to reduce opioid use. JOURNAVX is especially relevant for acute pain scenarios such as post-surgical recovery and injury care. A closeup of pills in a pharmacy, representing the high quality medications of the company. Beyond acute pain, Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) continues to expand its reach. Its next-gen cystic fibrosis therapy, ALYFTREK, received approval from the European Commission in July 2025, strengthening its global leadership in this area. The company is also advancing gene-editing therapies like CASGEVY for sickle cell and beta thalassemia, and is developing treatments for kidney disease, type 1 diabetes, and rare genetic disorders. While we acknowledge the potential of GOOGL as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None. Sign in to access your portfolio

Vertex Pharmaceuticals Incorporated (VRTX) Launches JOURNAVX, First Non-Opioid Acute Pain Treatment
Vertex Pharmaceuticals Incorporated (VRTX) Launches JOURNAVX, First Non-Opioid Acute Pain Treatment

Yahoo

time5 days ago

  • Business
  • Yahoo

Vertex Pharmaceuticals Incorporated (VRTX) Launches JOURNAVX, First Non-Opioid Acute Pain Treatment

We recently compiled a list of Vertex Pharmaceuticals Incorporated stands second on our list and has recently launched the first non opioid acute pain treatment. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX), a global biotech firm based in Boston, is known for developing innovative treatments for serious diseases like cystic fibrosis, sickle cell disease, and beta thalassemia. The company is recognized for its strong pipeline targeting unmet medical needs. In July 2025, Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) launched JOURNAVX (suzetrigine), a first-in-class, non-opioid treatment for moderate-to-severe acute pain. JOURNAVX, an oral NaV1.8 pain signal inhibitor, was named a 2025 Breakthroughs Innovation Celebration winner by Premier, Inc. It represents the first new class of pain medicine in decades and offers a safer alternative to opioids by directly targeting pain pathways without addiction risks. This launch marks a major advancement in pain management, aligning with public health efforts to reduce opioid use. JOURNAVX is especially relevant for acute pain scenarios such as post-surgical recovery and injury care. A closeup of pills in a pharmacy, representing the high quality medications of the company. Beyond acute pain, Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) continues to expand its reach. Its next-gen cystic fibrosis therapy, ALYFTREK, received approval from the European Commission in July 2025, strengthening its global leadership in this area. The company is also advancing gene-editing therapies like CASGEVY for sickle cell and beta thalassemia, and is developing treatments for kidney disease, type 1 diabetes, and rare genetic disorders. While we acknowledge the potential of GOOGL as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: The Best and Worst Dow Stocks for the Next 12 Months and 10 Unstoppable Stocks That Could Double Your Money. Disclosure: None.

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